Resumen de: WO2025162452A1
A compound specifically binding to an α-synuclein aggregate, and a preparation method therefor and the use thereof. Specifically, the compound binding to the α-synuclein aggregate comprises compounds as shown in formula A or sub-general formulas thereof, or stereoisomers, pharmaceutically acceptable salts, solvates or stable isotope variants thereof. The compound is a small molecule tracer, which can specifically recognize the α-synuclein aggregate, and can be used for the preparation of a drug for the treatment or diagnosis of neurodegenerative diseases (such as Parkinson's disease, dementia with Lewy bodies, multiple system atrophy, Alzheimer's disease, amyotrophic lateral sclerosis, progressive supranuclear palsy and progressive muscular atrophy) related to the α-synuclein aggregate and other misfolded proteins.
Resumen de: WO2025162103A1
The present invention relates to a C-17 carbonyl-substituted oleanane triterpene derivative, a preparation method therefor, and a use thereof. Provided are a C-17 carbonyl-substituted oleanane triterpene derivative, a use of the compound in the preparation of an NRF2 activator, and preparation of a medicament for treating/preventing diseases. The diseases comprise cerebral small vascular disease, mitochondrial encephalomyopathy, autism spectrum disorder, Rett syndrome, Friedreich ataxia, stroke, hemorrhagic cerebral apoplexy, ischemic cerebral apoplexy, multiple sclerosis, amyotrophic lateral sclerosis, schizophrenia, schizophrenic cognitive impairment, Parkinson's disease, cognitive impairment in Parkinson's disease, Alzheimer's disease, vascular dementia, epilepsy, Huntington's disease, heart failure, myocardial infarction, renal failure, kidney ischemia, etc., or other disease states and conditions which are obvious to a person skilled in the art. Also provided are a prodrug thereof, or a pharmaceutically acceptable salt thereof, or a hydrate or solvate thereof, and a pharmaceutical composition containing same.
Resumen de: WO2025162101A1
A C17-site nitrogen-substituted and methylene-substituted oleanane triterpene derivative, a preparation method therefor, and a use thereof. Provided are a C17-site nitrogen-substituted and methylene-substituted oleanane triterpene derivative, a use of the compound in the preparation of an NRF2-Leap1 decoupling agent, and a use of the compound in the preparation of a medicament for preventing and/or treating diseases in a patient, the diseases comprising cerebral small vessel disease, mitochondrial encephalomyopathy, autism spectrum disorder, Rett syndrome, Friedreich ataxia, stroke, hemorrhagic cerebral apoplexy, ischemic cerebral apoplexy, multiple sclerosis, amyotrophic lateral sclerosis, schizophrenia, cognitive impairment in schizophrenia, Parkinson's disease, cognitive impairment in Parkinson's disease, Alzheimer's disease, vascular dementia, epilepsy, Huntington's disease, heart failure, myocardial infarction, renal failure, kidney ischemia, etc.
Resumen de: WO2025162358A1
Disclosed herein is a compound of Formulas (I) and (II) as an FTO inhibitor with novel structures. Also disclosed herein is a pharmaceutical composition comprising the same, and a method of inhibiting weight gain, promoting weight loss, reducing serum LDL, cholesterol, LDL-c, or triglycerides, or treating obesity or an obesity-related disease (esp. obesity-related diabetes, hyperglycemia, diabetic nephropathy, hyperlipemia, coronary heart disease, atherosclerosis, hypertension, cardiovascular or cerebrovascular disease) or Alzheimer's disease by inhibiting FTO by using the compound disclosed herein.
Resumen de: WO2025161144A1
The present invention provides a novel Kv1.3 channel (or Kv1.3) inhibitor, which can be used for preventing and/or treating Kv1.3 channel (or Kv1.3)-related diseases, including immune and inflammatory diseases, such as multiple sclerosis, inflammatory bowel disease, ulcerative colitis, Crohn's disease, rheumatoid arthritis, type I diabetes, psoriasis and asthma, spondylitis and periodontitis; and obesity, type 2 diabetes, renal fibrosis, Alzheimer's disease, and ischemic stroke.
Resumen de: AU2024209890A1
The disclosure is directed to novel ALPK1 inhibitors having the Formula (I), or a pharmaceutical acceptable salt, a stereoisomer, a tautomer, a stable isotopic variant, a prodrug, or a crystal form thereof. The disclosure is also directed to pharmaceutical composition comprising the novel ALPK1 inhibitors, and use thereof in treating inflammation related diseases, such as ROSAH syndrome, inflammatory bowel disease (IBD), NASH, gout, diabetes, chronic kidney disease, pancreatitis, Kawasaki disease, inflammatory skin diseases and neurodegenerative diseases including the Alzheimer's disease.
Resumen de: US2025248985A1
The invention relates generally to the treatment of Parkinson's Disease (PD), including Parkinson's Disease psychosis (PDP) with iloperidone.
Resumen de: US2025248960A1
The present invention provides methods of providing rapid relief of motor fluctuations in a Parkinson's disease patient. The methods of the invention comprise pulmonary administration of levodopa by inhalation at therapeutically effective concentrations such that the patient's plasma levodopa concentration increases by at least about 200 ng/ml within 10 minutes or less post inhalation as compared to the concentration of levodopa in the patient's plasma prior to inhalation of the levodopa and wherein the patient's plasma concentration remains increased by at least about 200 ng/ml for a time period of at least 15 minutes after inhalation. The methods of the invention are particularly useful for treatment of motor fluctuations which arise as a result of poorly controlled levodopa plasma levels in a patient.
Resumen de: US2025250324A1
A fusion protein of the present disclosure may induce the degradation of neurodegenerative disease-causing factors by binding to Tau and amyloid-beta proteins as the neurodegenerative disease-causing factors and activating autophagy. In addition, the fusion protein of the present disclosure penetrates a blood-brain barrier and is introduced into cells to be effectively delivered into nerve cells without a separate carrier, and has high stability and thus is expected to be used as a platform for the treatment of neurodegenerative diseases such as dementia and Alzheimer's disease.
Resumen de: AU2025205635A1
This invention provides with an edaravone suspension for oral administration having excellent bioavailability. It is expected that burden on ALS patients and care workers can be reduced thereby. This invention provides with an edaravone suspension for oral administration having excellent bioavailability. It is expected that burden on ALS patients and care workers can be reduced thereby. ul h i s i n v e n t i o n p r o v i d e s w i t h a n e d a r a v o n e s u s p e n s i o n f o r o r a l a d m i n i s t r a t i o n h a v i n g e x c e l l e n t u l b i o a v a i l a b i l i t y t i s e x p e c t e d t h a t b u r d e n o n p a t i e n t s a n d c a r e w o r k e r s c a n b e r e d u c e d t h e r e b y
Resumen de: AU2025205501A1
18808542_1 (GHMatters) P43228AU01 Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington’s disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington’s disease. Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease. 18808542_1 (GHMatters) P43228AU01 ul u l r o v i d e d
Resumen de: WO2025163129A1
The present disclosure provides for treating Parkinson ́s disease (PD) comprising administering acetyl-leucine or a pharmaceutically acceptable salt thereof to a subject in need thereof.
Resumen de: WO2025162734A1
The present invention relates to a food- or feedstuff composition comprising a B. subtilis strain and a Ginkgo biloba extract, such composition for use as a medicament as well as such composition for use in treating or preventing neurodegenerative diseases, in particular Alzheimer's disease as well as the use of such composition as a food supplement.
Resumen de: WO2025162163A1
Disclosed in the present invention is the use of a mesenchymal-stem-cell-derived intracellular nanovesicle in neuroprotection. Compared to a small extracellular vesicle with exosomes as the main component, the small intracellular nanovesicle of the present invention has a smaller particle size, a narrower particle size distribution range, and greater stability at different temperatures, and has good tissue compatibility. The small intracellular nanovesicle of the present invention can better ameliorate nerve injury or neurodegenerative diseases such as optic nerve injury, ischemic stroke and Alzheimer's disease, and has very good application and research value in the field of pharmaceuticals.
Resumen de: CN120051284A
Provided herein are methods of treating early Alzheimer's disease using hydroxypropyl beta-cyclodextrin compositions.
Resumen de: EP4595956A1
A levodopa derivative including a compound or pharmaceutically acceptable salt, hydrate, and/or solvate thereof, wherein the compound includes substituents which, in aggregate, contain at least 6 carbon atoms which are only bonded to either other carbon atoms or to hydrogen atoms. The levodopa derivative may be formulated as a composition including one or more pharmaceutically acceptable carriers or excipients. The levodopa derivative may be part of a pharmaceutical composition including micro or nano particles in which the levodopa derivative is encapsulated in the pharmaceutically acceptable polymer. The levodopa derivative can be used to treat Parkinson's disease by administering to a mammal an amount sufficient to treat Parkinson's disease.
Resumen de: MX2025005198A
4-Aminopyrrolo2,I-f1,2,4triazine compounds of formula I for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4- aminopyrrolo2,1-fl,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).
Resumen de: MX2025007590A
Provided herein a method of treating Juvenile Huntington disease in a subject in need thereof comprising orally administering a pharmaceutical composition comprising pridopidine and/or its analog or a pharmaceutically acceptable salt thereof.
Resumen de: MX2025008034A
The present disclosure provides anti-amyloid β (Aβ) antibodies and antibody fragments that preferentially bind soluble amyloid Aβ protofibril/oligomer and trigger ADPC in microglial cells, anti-amyloid β (Aβ) antibodies and antibody fragments that reduce soluble amyloid Aβ protofibril/oligomer levels and insoluble amyloid Aβ plaque in brain tissue, and the use of anti-Aβ protofibril/oligomer antibodies and antibody fragments in therapy, prophylaxis, diagnosis, screening, and monitoring of conditions associated with Aβ protein aggregation, in particular Alzheimer's disease (AD).
Resumen de: MX2025008267A
The disclosure relates to compositions and methods for altering, <i>e.g.</i>, enhancing, the expression of GCase proteins, whether <i>in vitro</i> and/or <i>in vivo</i>. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.
Resumen de: US2025241950A1
Provided are chimeric antigen receptor (CAR) that bind to beta amyloid, macrophages (CAR-Ms) that express the CAR, and compositions comprising the same. Also provided are methods for reducing one or more symptoms associated with Alzheimer's disease using the CAR-Ms.
Resumen de: WO2025159427A1
The present invention relates to: an apolipoprotein E (APOE) antisense oligonucleotide; and a pharmaceutical composition for treating Alzheimer's disease comprising same. The antisense oligonucleotide of the present invention can reduce the expression of the APOE4 variant gene that contributes to increased risk and exacerbation of Alzheimer's disease, and can be used as an RNA therapeutic agent for diseases caused by abnormal levels of APOE proteins or APOE variant genome expression.
Resumen de: WO2025160116A1
A method includes treatment of a sporadic ALS patient with oral fasudil at a dose exceeding 240 mg/day. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.
Resumen de: WO2025159459A1
The present invention relates to a pharmaceutical composition for preventing or treating degenerative brain diseases, the composition comprising prasugrel as an active ingredient. The composition containing prasugrel or a salt thereof according to the present invention has excellent neuroprotective activity and, in particular, can suppress MPP+-induced neurotoxicity and neuronal cell death, suppress LPS-induced microglial cell activation and cell migration, and has neuroinflammation inhibitory activity, and thus has the effect of preventing, alleviating, and treating various degenerative brain diseases including Parkinson's disease.
Resumen de: US2025243269A1
The present disclosure provides compositions and methods for treating neurodegenerative diseases, in particular, Alzheimer's Disease, by using anti-FSH antibodies in a subject in need thereof. In some embodiments, the subject has a condition in which FSH levels are elevated. The methods include administering to said subject a therapeutically effective amount of an anti-FSH antibody or an antigen-binding portion thereof.
Resumen de: US2025243172A1
NLRP3 selective inhibitors (NSIs) as anti-inflammatory agents are provided, as are methods of using NSIs to inhibit inflammation and prevent or treat diseases and conditions associated with inflammation, such as Alzheimer's disease, Parkinson's disease, multiple sclerosis, traumatic brain injury, acute myocardial infarction, heart failure, arthritis, diabetes, gout, COVID-19, and autoinflammatory diseases.
Resumen de: AU2024208984A1
The present invention relates to a liposomal composition for use in a method of treating Parkinson's disease. The liposomal composition comprises sphingomyelin in a lipid bilayer and a therapeutically ef fective amount of monosialotetrahexosylganglioside (GM1), wherein a therapeutically ef fective dose of said liposomal composition is administered at most every 4 days in a primary mode of administration with at least 3 days between each administration; preferably at most every 6 days in a primary mode of administration with at least 5 days between each administration; most preferably at most every 7 days in a primary mode of administration with at least 6 days between each administration.
Resumen de: US2025237652A1
Provided herein are compositions and methods related to the production and detection of a histone H1.0 protein dimethylated at lysine residue 180 (K180) (H1.0K180me2 protein) or a histone H1.0 peptide dimethylated at a lysine residue corresponding to K180 (H1.0K180me2 peptides). The H1.0K180me2 protein and H1.0K180me2 peptides are useful for applications including, but not limited to, molecular diagnostics of DNA damage, genotoxic stress, radiation exposure, and Alzheimer's disease, therapeutics, monitoring of therapeutic regimens, patient stratification, and drug screening. Also provided herein are antibodies specific for the H1.0K180me2 protein and H1.0K180me2 peptides.
Resumen de: WO2025153721A1
The current invention relates to PLA2G15 inhibitors represented by formula (VI), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, HIV, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
Resumen de: WO2025153832A1
8Z, 11Z, 14Z, 17Z-eicosatetraenoic acid (ETA) and/or 10Z, 13Z, 16Z-docosa-10,l 3, 16-trienoic acid (DTA) have been shown to have anti-neuroinflammatory properties and suitable for use in the treatment of neurodegenerative disease, such as Alzheimer's disease. The anti-neuroinflammatory effect of using ETA and/or DTA can be surprisingly, and optionally synergistically increased by using ETA and/or DTA in combination with eicosapentaenoic acid (EPA),docosahexaenoic acid (DHA), stearidonic acid (6, 9, 12, 15 -octadecatrienioc acid) (SDA), gamma linolenic acid (6, 9, 12-octadecatrienioc acid) (GLA), dihomo γ linolenic acid (8, 11, 14-eicosatraenoic acid) (DGLA), and/or 7, 10, 13, 16, 19-docosapentaenoic acid (DPA), preferably docosahexaenoic acid (DHA).
Resumen de: WO2025153719A1
The current invention relates to PLA2G15 inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
Resumen de: WO2025153715A1
The current invention relates to PLA2G15 inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
Resumen de: WO2025152934A1
Provided herein is a method for treating Alzheimer's disease. The method comprises orally administering to a subject in need thereof 50-100 mg/day of orelabrutinib. The method reduces neuroinflammation and improves the cognitive functions such as learning and memory processes of the subject.
Resumen de: WO2025153720A1
The current invention relates to PLA2G15 inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
Resumen de: WO2025153718A1
The current invention relates to PLA2G15 inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, HIV, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
Resumen de: WO2025154076A1
This invention provides a method of prolonging the survival of subjects afflicted with ALS by administering a composition comprising pridopidine or pharmaceutically acceptable salt thereof.
Resumen de: US2025235464A1
Described herein are methods for inhibiting generation of one or more non-classical variant(s) of amyloid precursor protein (APP) gene. Provided herein are methods for diagnosing an individual having or suspected of having Alzheimer's disease following identification of an expression profile or an activity profile of the one or more non-classical variant(s) and treating the individual using a reverse transcriptase inhibitor or salt thereof.
Resumen de: WO2025152110A1
A drug combination for treating Alzheimer's disease and a pharmaceutical composition thereof. The pharmaceutical composition comprises: (a) a prophylactically or therapeutically effective amount of HDAC6 inhibitor; and (b) a prophylactically or therapeutically effective amount of GSK-3β inhibitor. The components of the drug combination are used in combination, so that the therapeutic effect of each single drug on Alzheimer's disease can be synergistically enhanced. Moreover, significant weight loss or abnormal behavior does not appear in mice after drug administration, showing that the drug combination has good efficacy and safety.
Resumen de: WO2025155903A1
The present invention provides compositions and methods for treating disorders of the central nervous system. In some embodiments, compositions of the present invention comprise novel compounds further comprising a heterocyclic core optionally fused with a 6-membered carbocyclic ring and a non-electrophilic substituent. In one embodiment, the disorder of the central nervous system which can be treated using the present invention is Alzheimer's disease.
Resumen de: WO2025154692A1
Provided is a novel means capable of efficiently inhibiting beta-secretase (BACE1). Specifically, a BACE1-inhibiting composition containing a specific glycerophospholipid is provided.
Resumen de: AU2023341167A1
The present disclosure relates to methods of treating Alzheimer's disease, diseases and/or conditions associated with Covid-19 infection, including long COVID, a post-acute infection syndrome, or symptoms of orthostatic intolerance comprising administration of a therapeutically-effective combination of a COX-2 inhibitor and an antiviral compound.
Resumen de: KR20250111720A
본 발명은 카나비크로멘산 및 아자인돌 유도체의 신규한 용도인 파킨슨병의 예방 또는 치료 용도를 제공한다. 본 발명에서 사용된 카나비크로멘산 및 아자인돌 유도체 화합물은 파킨슨병의 주요 증상인 운동기능 장애에 대한 개선 효능을 나타냄으로써, 이들 카나비크로멘산, 및 특정 구조의 아자인돌 유도체가 우수한 인지기능 개선 효과를 나타낸다는 것을 확인하였다. 따라서, 카나비크로멘산 및 아자인돌 유도체는 의약 및 식품 분야에서 파킨슨병에 따른 인지기능 및 운동기능 저하 증상의 완화, 예방 또는 치료 용도로 유용하게 사용될 수 있다.
Resumen de: WO2025151408A1
Described are Microtubule-associated protein tau (MAPT) antisense oligonucleotides (ASOs) and MAPT ASO conjugates, and methods of using the MAPT ASOs and MAPT ASO conjugates to treat neurodegenerative disorders, such as Alzheimer's disease.
Resumen de: US2025230172A1
Provided herein are compounds that modulate glucocerebrosidase (GCase), an enzyme whose activity is associated with neurological diseases and disorders (e.g., Gaucher's disease, Parkinson's disease). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating GCase-related diseases and disorders (e.g., Gaucher's disease, Parkinson's disease) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Resumen de: US2025228868A1
The present invention relates to the treatment of a sporadic ALS patient with oral fausdil at a dose of 180-240 mg/day. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.
Resumen de: US2025230149A1
The present disclosure provides compositions of (4R,5R)-5-(2-chlorophenyl)-4-(5-(phenylethynyl)pyridin-3-yl)oxazo-lidin-2-one (Compound 1). Crystalline and solvate forms of the compound and formulations comprising the compound are also provided. Methods of using the compound and methods of administering the formulations to a subject in need thereof are provided to treat or prevent CNS disorders such as Alzheimer's disease.
Resumen de: US2025230206A1
Inhibitors are provided for targeting γ-secretase to reduce amyloid load as a viable strategy in Alzheimer's disease treatment and drug discovery. γ-secretase has been shown to cleave amyloid precursor protein, causing an increase in the extracellular concentration of amyloid-β peptides. This extracellular concentration increase can lead to a build-up of amyloid plaques in patients and associated health complications for them. The inhibitors bind adjacent the transmembrane domain of amyloid precursor protein through both covalent and non-covalent interactions. These interactions inhibit the ability of γ-secretase to cleave the amyloid precursor protein, halting the build-up of extracellular amyloid plaques. The inhibitors exhibit specificity for amyloid precursor proteins, reducing concerns of potential off-target effects.
Resumen de: WO2025150507A1
To provide an insulin amyloid polymerized protein, an antibody, antibody-producing B cells and a medical composition capable of effectively preventing and treating Alzheimer type dementia with little side effects. An insulin amyloid polymerized protein is collected from a patient with Alzheimer type dementia to which insulin has been administered for use in the treatment of the patient. An insulin amyloid polymerized protein composition for use in the treatment of a patient with Alzheimer type dementia, wherein an insulin amyloid polymerized protein is obtained by chemically bonding insulin and amyloid. Also provided are an antibody therefor, antibody-producing B cells, and a medical composition.
Resumen de: WO2025151884A1
The present invention is directed to methods and compositions for the administration of sodium pentosan polysulfate and related glycosaminoglycans, particularly by oral administration, particularly in combination with administration of an intestinal penetration agent. The methods and compositions are suitable for treatment of neurodegenerative diseases such as, but not limited to, Alzheimer's disease. Methods and compositions according to the present invention can be used together with other agents suitable for treatment of neurodegenerative diseases such as, but not limited to, Alzheimer's disease.
Resumen de: WO2024054791A1
The disclosure relates to a combination of active ingredients/adjuvants for the treatment of neurological disorders and diseases such as Alzheimer's disease and mild cognitive impairment (MCI) and memory and cognitive disorders and conditions. In particular, combinations of scyllo-inositol and treatments for Alzheimer's disease such as aducanumab and/or combinations with essential fatty acids such as mixtures of linolenic acid/linoleic acid or vitamin D or vitamin D compounds such as calcifediol are disclosed as useful. The combinations may be in the form of separate dosage forms of each active ingredient or may be an oral dosage form having multiple active ingredients in a single capsule or tablet or oral solution. The invention also relates to a method of treating patients having mild cognitive impairment with MMSE criteria of between 22 to 26 with a pharmaceutically effective amount of scyllo-inositol to treat the disease and to slow down progression to Alzheimer's disease.
Resumen de: GB2637227A
A method of monitoring and treating a subject with ALS based on biomarkers. In some embodiments, the method comprises: identifying that a subject has a ratio of LPS1EGF associated with ALS; and based on the identifying that the subject has the ratio of LPS:EGF associated with ALS, determining that the subject is eligible for sodium chlorite therapy for the ALS, determining based on the ratio of LPS:EGF whether to continue the therapeutic regimen of sodium chlorite; wherein the ratio of LPS:EGF in the subject is no greater than 50 and the therapeutic regimen of sodium chlorite is about 0.2 mg/kg/day to about 3.5 mg/kg/day administered orally and/or parenterally.
Resumen de: AU2023400894A1
The present invention relates to the medical field, in particular, to a nanotechnological based Enzyme Replacement Therapy, preferably for Parkinson's disease, based on the restoration of lysosomal glucocerebrosidase activity through enzyme-polymer nanoconjugation of GBA, the GBA polymer conjugate for such use, and its manufacturing method.
Resumen de: US2025223290A1
Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, heart failure, idiopathic pericarditis, atopic dermatitis, inflammatory bowel disease, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Resumen de: US2025223589A1
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
Resumen de: US2025222072A1
Aspects of the disclosure relate to compositions and methods useful for treating Parkinson's disease. In some embodiments, the disclosure provides a method for treating Parkinson's disease comprising administration of a viral vector comprising a GDNF nucleic acid sequence. In some embodiments, administration is locally to the subject putamen. In some embodiments, administration is systemically, e.g., via the viral vector comprising a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
Resumen de: AU2023420087A1
Provided herein a method of treating Juvenile Huntington disease in a subject in need thereof comprising orally administering a pharmaceutical composition comprising pridopidine and/or its analog or a pharmaceutically acceptable salt thereof.
Resumen de: TW202434616A
Compounds, compositions, uses, and methods for increasing cell viability of a dopaminergic neuron, or for preventing or treating dopaminergic neuronal death, are provided herein. In certain examples, methods for reducing symptoms and/or for preventing or treating Parkinson's disease in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a<SP>GDP</SP>), one or more expressible nucleic acids encoding Rab1a<SP>GDP</SP>, or a combination thereof.
Resumen de: EP4582144A2
The invention relates to novel monoclonal anti-alpha-synuclein antibodies. The antibodies can be used for treating a synucleinopathy such as Parkinson's disease (including idiopathic and inherited forms of Parkinson's disease), Diffuse Lewy Body Disease (DLBD), Lewy body variant of Alzheimer's disease (LBV), Combined Alzheimer's and Parkinson disease, pure autonomic failure and multiple system atrophy.
Resumen de: EP4582412A1
The present invention relates to: a method for preparing a chiral gamma lactam derivative or a pharmaceutically acceptable salt thereof by using a chiral organocatalytic compound; and a composition for preventing, alleviating or treating muscle diseases, mental diseases, or neurodegenerative diseases, comprising the derivative or the pharmaceutically acceptable salt thereof. The chiral gamma lactam derivative or the pharmaceutically acceptable salt thereof, of the present invention, has an effect of inhibiting MAO-B and MSTN, targets D1-mClu5, and can be used in the prevention, alleviation, or treatment of muscle diseases including sarcopenia, mental diseases including depression, neurodegenerative diseases including Parkinson's disease, and the like.
Resumen de: WO2025141029A1
Disclosed are compounds of formula (I), or a pharmaceutically acceptable salt thereof. Also disclosed are a medicament and a pharmaceutical composition comprising said compounds of formula (I), and said compounds (I) for use in the treatment of a neurodegenerative disease such as Parkinson's disease. Further disclosed are a solid form of a compound of Formula (I-a), characterized as crystalline Form A, as well as a pharmaceutical composition comprising said solid form, and said solid form for use in treating a neurodegenerative disease.
Resumen de: WO2025141063A1
The present invention relates to Phosphatidylinositol 3,5-bisphosphate (PI(3,5)P2) of formula (I), preferably formulated in LNP, preferably SANP, and optionally together with a miRNA or antagomir, for use in the treatment of neurodegenerative disorders, like AD, PD and ALS, characterized by impaired/blocked autophagy. It also relates to a LNP, preferably SANP, containing PI(3,5)P2 and optionally a miRNA or antagomir.
Resumen de: WO2025143100A1
The present invention addresses the problem of providing a medicine that, despite of being a non-ergot dopamine agonist (DA), reduces the risk of the drowsiness side effect, and exhibits an excellent therapeutic effect on neurodegenerative disorders such as Parkinson's disease (PD). The present invention relates to a pharmaceutical composition for treating neurodegenerative diseases such as Parkinson's disease, the pharmaceutical composition comprising 1-{(4aR,6R,8aR)-2-amino-3-cyano-8-methyl-4,4a,5,6,7,8,8a,9-octahydrothieno3,2-gquinolin-6-ylcarbonyl}-3-2-(dimethylamino)ethyl-1-propylurea or a pharmacologically acceptable salt thereof. The pharmaceutical composition is characterized by being orally administered at a daily dose of 0.25 mg to 2 mg in terms of free form.
Resumen de: AU2023408627A1
The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).
Resumen de: US2025213507A1
Tramiprosate and derivatives thereof are provided herein for treating neurodegenerative disorders such as Alzheimer's disease (AD).
Resumen de: US2025213619A1
A composition for treating intractable neurological diseases contains culture supernatant obtained by culturing deciduous dental pulp stem cells in serum-free medium under ultrasound irradiation. A pharmaceutical product for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer's disease contains the composition for the treatment of intractable neurological diseases. In a method for producing a composition for treating intractable neurological diseases, the composition contains a culture supernatant obtained by culturing deciduous dental pulp stem cells under ultrasound irradiation using serum-free medium.
Resumen de: US2025213622A1
The use of extract from rabbit skin inflamed by vaccinia virus for treating Parkinson's disease or restoring neurological function of the brain or alleviating damage to neurological function of the brain in a patient suffering from Parkinson's disease is provided. The extract from rabbit skin inflamed by vaccinia virus can be Lepalvir.
Resumen de: US2025213564A1
Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).
Resumen de: US2025213611A1
The present disclosure provides, among other things, methods for the treatment of neurodegenerative diseases (ND) and other mitochondrial disorders, and compositions related thereto. Described herein are in vitro (cell culture) and in vivo (animal model) experimental examples demonstrating mitochondrial organelle transplantation (MOT) for the treatment of NDs such as amyotrophic lateral sclerosis (ALS). Furthermore, as discussed herein, MOT has been performed in five human ALS patients with positive results—measurable improvement of their conditions has been observed, with no adverse events.
Resumen de: US2025214948A1
There is provided herein a compound of formula I,wherein R1, R2, n, X, Q, L, m, R3 and p are as defined herein, which compounds are useful in the treatment of treatment of diseases characterised by impaired signalling of neurotrophins and/or other trophic factors, such as Alzheimer's disease and the like.
Resumen de: AU2024208288A1
Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression into synucleinopathies and Parkinson's disease by administering a formulation comprising an effective amount of piromelatine to the subject. Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression of parasomnias into chronic post-traumatic stress disorder (PTSD) by administering a formulation comprising of an effective amount of piromelatine to a subject.
Resumen de: AU2025204414A1
Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease. Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
Resumen de: AU2025204524A1
Abstract The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT- containing dosage units and other compositions. Abstract The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT- containing dosage units and other compositions. un b s t r a c t u n h e i n v e n t i o n p r o v i d e s n o v e l d o s i n g r e g i m e n s f o r e u c o - e t h y l t h i o n i n i u m ( ) c o m p o u n d s w h i c h m a x i m i s e t h e p r o p o r t i o n o f s u b j e c t s i n w h i c h t h e c o n c e n t r a t i o n w i l l e x c e e d c o n c e n t r a t i o n s i n w h i c h t h e r a p e u t i c e f f i c a c y i n r e l a t i o n t o t r e a t m e n t o f n e u r o d e g e n e r a t i v e d i s o r d e r s s u c h a s l z h e i m e r ' s d i s e a s e a n d r o n t o t e m p o
Resumen de: US2025214984A1
Disclosed are compounds of following formula (I):in which all symbols have the same meanings as the definitions described in the specification; or a salt thereof. The compounds or a salt thereof are useful as a prophylactic and/or therapeutic agent for cancer, Huntington's disease, Alzheimer's disease and the like.
Resumen de: EP4578461A2
Methods and pharmaceutical unit dosage forms for treating Parkinson's disease in a subject (e.g., an "off" episode in a subject having Parkinson's disease) are described. The pharmaceutical unit dosage forms are films having a first portion including particles containing an acid addition salt of apomorphine and a second portion containing a pH neutralizing agent. The pharmaceutical unit dosage forms can be flexible and have toughness greater than 100 g × mm. The methods can involve administering to a subject having Parkinson's disease a therapeutic dose sufficient to produce an apomorphine plasma concentrate of at least 2.64 ng/mL within 45 minutes after the administration. The subject may be identified as having low uptake, medium uptake, or high uptake of apomorphine administered via oral mucosa.
Resumen de: AU2023330511A1
The present disclosure relates to an sgRNA and its application in the preparation of a product for the treatment of Huntington's disease. The present disclosure was designed and screened to obtain an sgRNA targeting exon 1 of the human HTT gene as shown in SEQ ID NO: 1 or SEQ ID NO: 2. The CRISPR/Cas9 system mediated HTT gene knockout strategy based on this sgRNA and its high homologue sgRNA can efficiently knock out the human Huntingtin gene and achieve gene therapy for Huntington's disease.
Resumen de: WO2024044355A2
Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).
Resumen de: MX2025004703A
Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl) sulfonyl)-4, 4a, 5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl) (pyridin-2-yl)methanone, having the chemical structure illustrated as. Suitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.
Resumen de: MX2025007343A
The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).
Resumen de: MX2025006917A
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
Resumen de: MX2025003095A
The present disclosure provides compounds of Formula I: (I), or an N- oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: A, Y, m, n, p, R1, R2, R3, R3a, R4, R5, R6, R7, and Z are as described herein; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds, N-oxides, or salts, and their uses for treating M4-mediated (or M4-associated) disorders including, e.g., Alzheimer's Disease, Parkinson's Disease, schizophrenia (e.g., its cognitive and negative symptoms), pain, addiction, and a sleep disorder.
Resumen de: MX2025001192A
Provided is a drug for treating Alzheimer's disease, the drug enabling retention of cognitive function amelioration and nerve quality improvement for a specific time even after treatment ends.ã¿¿This drug for causal treatment of Alzheimer's disease (disease-modifying drug) contains hydrogen gas as an active ingredient.
Resumen de: WO2025134068A1
The invention provides antibody conjugate compositions comprising an antibody targeting a pathological protein covalently attached to one or more brain penetrant, pathological protein binding small molecule entities by a linker. The invention further provides methods of treating neurodegenerative diseases and disorders such as Alzheimer's disease with the antibody conjugate compositions.
Resumen de: WO2025137077A1
Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells. The methods of treatment involve the administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the efficacy of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive function and/or quality of life.
Resumen de: AU2025204068A1
Abstract Provided herein are sulfopropanoic acid derivatives or pharmaceutically acceptable salts thereof, for treating a disease characterized by amyloid and amyloid-like aggregates, e.g., Alzheimer's disease.
Resumen de: US2025205239A1
The present invention provides a composition for preventing or treating a neurodegenerative disease containing a phosphodiesterase 5 inhibitor (PDE5 inhibitors) and an acetylcholinesterase inhibitor (AChEI) and a method using thereof, wherein the PDE5 inhibitor is selected from among mirodenafil, sildenafil, vardenafil, tadalafil, udenafil, dasantafil, avanafil, pharmaceutically acceptable salts, solvates, hydrates, and a mixture thereof; and the AchEI is selected from among donepezil, rivastigmine, galantamine, physostigmine, tacrine, metrifonate, phenserine, tolserine, eseroline, huperizine A and B, galangin, cardanol, donepezil-AP2238, donepezil-tacrine, tacrine-ferulic acid hybrid, tacrine-hydroxyquinoline, ladostigil, indenyl derivatives, pharmaceutically acceptable salts, solvates, hydrates and a mixture thereof; and the neurodegenerative disease is dementia, Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD) or Multiple sclerosis (MS).
Resumen de: US2025205368A1
The present invention relates to Adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. Use of the methods and materials is indicated. for example. for treatment of lower motor neuron diseases such as SMA and ALS as well as Pompe disease and lysosomal storage disorders.
Resumen de: US2025205281A1
The present invention relates to a pharmaceutical composition for preventing and treating a degenerative brain disease, specifically dementia, comprising an activated natural killer cell as an active ingredient. The pharmaceutical composition comprising a mouse-derived activated natural killer cell as an active ingredient, according to the present invention, regulates the activity of microglial cells and inhibits the deposition of amyloid β plaques. In addition, the pharmaceutical composition showed an excellent effect on cognitive function improvement in an animal model of dementia. Furthermore, when a human peripheral blood mononuclear cell-derived natural killer cell and a human induced pluripotent stem cell-derived natural killer cell were activated, it was confirmed that the expression of some genes involved in restoring the activity of microglial cells was similar to or higher than that of a mouse-derived activated natural killer cell. Therefore, the pharmaceutical composition of the present invention can be effectively used for preventing or treating a degenerative brain disease, such as dementia, Parkinson's disease, and Huntington's disease, and improving cognitive impairment.
Resumen de: US2025205321A1
The present invention relates to therapeutical uses of non-classical human major histocompatibility complex (MHC) molecules (also named MHC class Ib molecules) in combination with peptide antigens for the treatment of Parkinson's disease. The invention more specifically relates to recombinant polypeptides comprising peptide antigens and one or more domains of a non-classical MHC class Ib molecule. The invention also relates to methods of producing such recombinant polypeptides, pharmaceutical compositions comprising the same, as well as their uses for treating Parkinson's disease.
Resumen de: US2025205305A1
The present disclosure provides to systems, methods, and compositions for rescuing protein misfolding and preventing protein aggregation. Particularly the present disclosure provides methods and compositions comprising DNAJB6 or variants thereof, or polynucleotides encoding DNAJB6 or variants thereof. The present disclosure also provides methods for treating protein misfolding and/or protein aggregation diseases (e.g., multiple amyotrophic lateral sclerosis and frontotemporal dementia) by administering the systems or compositions to a subject in need thereof.
Resumen de: WO2025130951A1
The present invention provides crystalline forms of 6-2-1-(2-pyridylmethyl)-4-piperidylethylspiro1,3dioxolo4,5-fisoindole-7,1'-cyclopropane-5-one phosphate (AD-35), and preparation methods therefor. The invention further provides a use of the crystalline forms of AD-35 in the preparation of a drug for treating Alzheimer's disease.
Resumen de: WO2025136898A1
The present disclosure is directed to compounds of Formula (I) and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.
Resumen de: WO2025136887A1
The present disclosure describes compounds and methods for disrupting the amyloid cascade.
Resumen de: WO2025136936A1
The present disclosure is directed to compounds of Formula I and their use as TREM2 agonists for treatment and prevention of a neurodegenerative disorder associated with a loss of function of human TREM2. The disclosed TREM2 agonists may be useful for the treatment of Alzheimer's Disease and associated neurological conditions.
Resumen de: WO2025131275A1
The present invention relates to pharmaceutical compositions and medicaments comprising the compound of formula (I); or a stereoisomer, tautomer, pharmaceutically usable solvate or salt thereof, and dosage regimens for the administration thereof to human patients.
Resumen de: WO2025137210A1
The present disclosure relates to a compound of formula (1) as an anhydrate which is in a crystalline Form 1, characterized by having a powder-X-ray diffractogram displaying a peak expressed as degree 2-Theta angles at about 8.3 and a solid form thereof. The present disclosure also relates to processes for its preparation, as well as a medicament and a pharmaceutical composition comprising it. The present disclosure further concerns the anhydrate crystalline Form 1 of compound of formula (1) for use as a medicine and more particularly in the treatment of Alzheimer disease, multiple sclerosis, and amyotrophic lateral sclerosis (ALS).
Resumen de: EP4573926A1
The present invention relates to a composition for preventing, ameliorating, or treating Parkinson's disease comprising osmotin protein as effective component, and, by having the effects of alleviating behavior and motor skill deficits induced by MPTP/α-synuclein, protecting dopaminergic neurons, regulating the expression level of neuroinflammation-related proteins, inhibiting apoptotic cell death induced by MPTP/α-synuclein, alleviating cell damage caused by overexpression of α-synuclein (A53T), reducing the accumulation of α-synuclein caused by activation of AMPK and subsequent autophagy, regulating the dendritic complex structure, increasing the spine density in pyramidal neurons, alleviating the cognitive deficits, and restoring the expression of synaptic markers, the osmotin protein can be advantageously used for a functional health food or a pharmaceutical product for preventing, ameliorating, or treating Parkinson's disease.
Resumen de: CN119907664A
A method of treating Parkinson's disease in a patient that accepts N doses per day of levodopa to provide X mg total daily dose of levodopa and that begins to experience motion fluctuations or begin to show a sign of "hypoefficacy", the treatment comprises administering more than N doses per day of levodopa to provide X mg of a total daily dose of levodopa, and administering Y mg of opirapone in a single daily dose, where X is from 100 to 1000, N is from 2 to 10, and Y is from 25 to 50.
Resumen de: US2025195457A1
Disclosed is a pharmaceutical composition for treatment of Parkinson's disease comprising N-(1S)-2,2,5,7-tetrafluoro-2,3-dihydro-1H-inden-1-ylsulfamide represented by formula (1) or a pharmaceutically acceptable salt thereof.
Resumen de: US2025195511A1
Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control, and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, osteoarthritis, atherosclerosis, heart failure, idiopathic pericarditis, myocarditis, atopic dermatitis, hidradenitis suppurativa, inflammatory bowel disease, cancer, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Resumen de: JP2025023319A
To provide compositions and methods for treating and preventing amyotrophic lateral sclerosis.SOLUTION: Dosage regimens for SOD1-targeting antisense oligonucleotides and salts thereof are provided. These dosage regimens find use in the treatment of subjects having or at risk of developing amyotrophic lateral sclerosis. In a first aspect, the disclosure provides a method of treating or preventing amyotrophic lateral sclerosis associated with a mutation in the human superoxide dismutase 1 (SOD1) gene in a human subject in need thereof.SELECTED DRAWING: None
Resumen de: AU2023403437A1
The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.
Resumen de: WO2025128781A1
Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, heart failure, idiopathic pericarditis, atopic dermatitis, inflammatory bowel disease, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Resumen de: US2025197862A1
Provided herein are artificial microRNA (miRNA) molecules for treating tauopathies. In some embodiments, the miRNA molecules target expression of Tau protein. Further provided herein are expression constructs, vectors (e.g., rAAV), cells, viral particles, and pharmaceutical compositions containing the artificial miRNA molecules. Yet further provided herein are methods and kits related to the use of the miRNA molecules, for example, to treat tauopathies including is Alzheimer's disease, progressive supranuclear palsy, corticobasal degeneration, frontotemporal dementia with parkinsonism-17, Pick's Disease, argyrophilic grain disease, globular glial tauopathy, chronic traumatic encephalopathy and post-encephalitic parkinsonism.
Resumen de: US2025195554A1
A gene expression regulator containing microparticles containing miRNA which targets a gene related to the expression of at least one kind of protein of amyloid precursor protein (APP), β-secretase (BACE1), an NMDA-activating protein, glycogen synthase kinase-3β (GSK-3β) and polyglutamine-binding protein-1 (PQBP1) can provide a novel gene expression regulator which can regulate (suppress, inhibit or the like) the expression of a protein related to amyloid β-related or tau protein-related dementia or brain inflammation such as Alzheimer's disease; an agent for preventing or treating Alzheimer's disease; and a method for improving dementia.
Resumen de: US2025197375A1
Disclosed are compounds comprising pyrimidinyl core and pharmaceutical compositions useful in the treatment of neurological disorders. The compounds described herein, alone or in combination with other pharmaceutically active agents, can be used for treating or preventing neurological diseases, including FTLD-TDP, chronic traumatic encephalopathy, ALS, Alzheimer's disease, LATE, or frontotemporal lobar degeneration.
Resumen de: WO2025128777A1
Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control, and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, osteoarthritis, atherosclerosis, heart failure, idiopathic pericarditis, myocarditis, atopic dermatitis, hidradenitis suppurativa, inflammatory bowel disease, cancer, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Resumen de: WO2025125071A1
Cathepsin D peptides are provided that are capable of interacting with and/or inhibiting the formation of beta amyloid aggregates, thereby leading to a competitive reduction of amyloid- amyloid interactions. Also provided are recombinant expression vectors encoding said peptides as well as of pharmaceutical formulations comprising said peptide-analogues. Said peptides, compositions and recombinant vectors are useful as therapeutic agents in the treatment and/or amelioration of the symptoms of amyloidoses such as Alzheimer's disease.
Resumen de: WO2025126158A2
SnRNA systems targeting SOD1 RNA sequences are disclosed herein. Further disclosed are methods of treating Amyotrophic Lateral Sclerosis.
Resumen de: WO2025128134A1
A composition and a method of treatment for treatment of diseases related to overexpression of SNCA gene in a subject such as Parkinson's disease comprising one or more clustered regularly interspaced short palindromic repeats associated protein (Cas9) or a variant thereof and one or more guide ribonucleic acids (RNAs). The present invention also provides a nanoparticle encapsulating any embodiment of the composition of the present invention.
Resumen de: EP4570798A1
The present invention relates to an aromatic heterocycle-fused cyclohexyl aminoalkyl piperidine derivative, a preparation method and use thereof. Specifically, the present invention provides a compound of general formula (I), a stereoisomer thereof, a tautomer thereof, or a pharmaceutically acceptable salt thereof, and a preparation method therefor, use thereof for activating the activities of a 5-HT<sub>1A</sub> receptor and dopamine D<sub>2</sub> and D<sub>3</sub> receptors, and use thereof in the preparation of a medicament for Parkinson's disease.
Resumen de: EP4570823A1
The present invention relates to an improved antibody specifically binding to amyloid-β oligomers (AβOs). Specifically, the present invention relates to an improved form of the antibody W20. Compared with the antibody W20, the improved form of the antibody W20 has a significantly improved affinity to AβOs, and can more significantly inhibit the aggregation of Aβ and the AβOs-induced toxicity of nerve cells, more effectively improve the cognition and memory functions of an Alzheimer's disease model mouse, and reduce pathological changes in the brain of the mouse. The improved form of the antibody can specifically bind to oligomers of an amyloid-β, α-synuclein, mHTT and SOD 1, can inhibit the aggregation and cytotoxicity of various amyloids, and has a better potential for treating various amyloid diseases, such as Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis, than the antibody W20. The improved form of the antibody can specifically bind to a highly toxic amyloid protein oligomer Aβo*3F, and has a better AD diagnosis value. The amino acid sequence of the antibody W20 is as shown in SEQ ID NO: 1.
Nº publicación: EP4570793A1 18/06/2025
Solicitante:
LUYE INNOMIND PHARMA SHIJIAZHUANG CO LTD [CN]
Luye Innomind Pharma Shijiazhuang Co., Ltd
Resumen de: EP4570793A1
The present invention relates to a new compound as a 5-HT<sub>2A</sub> receptor inverse agonist, a preparation method therefor and a pharmaceutical composition. The present invention further relates to the use of the compound or the pharmaceutical composition in the preparation of a drug for treating 5-HT<sub>2A</sub> receptor-related diseases. The diseases include non-motor symptoms caused by Parkinson's disease: delusions, hallucination, depression, anxiety, cognitive impairment and sleep disorders; dementia-related mental diseases; severe depression; or negative symptoms of schizophrenia, etc.