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151
results.
Updated on
03/10/2025 [07:32:00]
Absstract of: US2025304970A1
Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.
Absstract of: US2025306037A1
The present disclosure relates to methods useful to identify subjects having an increased risk for conversion to mild cognitive impairment (MCI) due to Alzheimer's disease (AD) and/or stage a subject prior to the onset of mild cognitive impairment (MCI) due to Alzheimer's disease (AD) and/or identify subjects with Aβ amyloidosis and/or to identify subjects who should or should not undergo further testing or treatment for Aβ amyloidosis, as well as methods for treating subjects diagnosed with Aβ amyloidosis by the methods disclosed herein.
Absstract of: AU2024220937A1
Provided herein are compounds, phannaceutical compositions, and methods of use for reducing the amount or activity of SNCA mRNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. Such compounds, pharmaceutical compositions, and methods of use are useful to ameliorate at least one symptom or hallmark of a synucleinopathy. Such synucleinopathies include Parkinson's disease, dementia with Lewy bodies (DLB), diffuse Lewy body disease, Parkinson's disease dementia (PDD), pure autonomic failure, multiple system atrophy (MSA), neuronopathic Gaucher's disease, and Alzheimer's disease.
Absstract of: AU2025230659A1
Abstract Provided herein are compounds that inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein. Abstract Provided herein are compounds that inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Absstract of: AU2024238511A1
The present disclosure relates to biomarkers and uses thereof in methods for selecting a patient diagnosed with amyotrophic lateral sclerosis (ALS) for an ALS therapy. The present disclosure further relates to methods for identifying the severity of ALS in a patient, treating an ALS patient, and monitoring efficacy of an ALS treatment.
Absstract of: EP4624448A1
A 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound and a preparation method and use thereof are provided, belonging to the field of drug development technology. The prepared 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound may be used for the preparation of an antiepileptic drug and drugs for treatment and/or prevention of traumatic craniocerebral injury disorders, ischemic stroke, hemorrhagic stroke, and Parkinson's disease.
Absstract of: WO2025199248A1
Applicants have discovered differential expression and activity of various miRNAs in neurodegenerative diseases. These miRNAs can serve as therapeutic targets for prevention and treatment of neurodegenerative diseases. Accordingly, embodiments of the invention include formulations and methods for treating neurodegenerative diseases such as dementia and Alzheimer's disease. The formulations and methods can include five microRNAs and two small molecule therapeutics. In aspects, the miRNAs are MIR-145-3p, let-7c-3p, MIR-383-5p, MIR-548aj-3p and MIR-548x-3p. In aspects, the small molecule therapeutics are GATC-49, fucosterol and fucoidan. In aspects, the compounds work synergistically with one another.
Absstract of: AU2024232317A1
The present invention provides a pharmaceutical composition comprising metformin and glibenclamide for use in the treatment of Parkinson's disease. The invention also comprises a combined administration of metformin and glibenclamide. In a preferred embodiment, the administration is made through oral route.
Absstract of: WO2025199451A2
Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells (MSCs). The methods of treatment involve an administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the effectiveness of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive or quality-of-life function.
Absstract of: WO2025199234A1
This disclosure relates to, inter alia, certain compounds, compositions, and pharmaceutical compositions thereof, that modulate the activity of the transporter protein vesicular monoamine transporter- 2 (VMAT2) and are directed to methods useful in the treatment of transporter protein vesicular monoamine transporter-2 mediated disorders, such as, neurological or psychiatric disease or disorders, including but not limited to, hyperkinetic movement disorders (e.g., tardive dyskinesia, Tourette's syndrome, Huntington's disease, tics, ataxia, chorea (such as, chorea associated with Huntington's disease), dystonia, hemifacial spasm, myoclonus, restless leg syndrome, and tremors). The disclosure further relates to synthetic methods and intermediates useful in the preparation of compounds.
Absstract of: WO2025199206A1
Provided herein are medical uses of CD11a-enriched T regulatory cells for treating amyotrophic lateral sclerosis (ALS) or ameliorating a symptom of ALS. Such uses may include longitudinal analysis of biomarkers, such as inflammatory cytokines.
Absstract of: WO2025199495A1
The present invention provides methods and compositions for reducing internalization and/or trafficking of tan in neuronal cells comprising contacting the cells with an effective amount of VLDL receptor antagonist. The invention further provides a method of treating or preventing Alzheimer's disease in a subject in need thereof, comprising administering to the subject an effective amount of a VLDL receptor antagonist.
Absstract of: US2025295603A1
Disclosed herein compositions and methods for the treatment of neurodegenerative disorders, such as Alzheimer's disease.
Absstract of: WO2025198916A1
The present disclosure provides anti-tau antibodies, including compositions and methods of using such antibodies for treating Alzheimer's disease.
Absstract of: WO2025194870A1
The present invention belongs to the technical field of biomedicine, and relates to a class of dexborneol or fenchyl alcohol ester derivatives of 2-hydroxynicotinic acid, and pharmaceutical uses thereof. The structural general formula of the derivatives is (1), R1 being selected from -H, -Cl, a hydrocarbon group having 1-8 carbon atoms, -CF3, -CF2H, -CN, -SCH3, -OH, -CHO, -CH2OH, -CH2NH2, -COOH, halogenated phenyl, or -NR4R5; R4, R5 are independently selected from -H or -CH3; R2 is selected from -H, an alkyl group having 1-6 carbon atoms, an acyl group having 2-7 carbon atoms, or a carbamoyl group; R 3 is selected from formula (a) or formula (b); X is selected from -CH= or -N=. The derivatives have good inflammation-inhibiting effects, and at high concentrations, they exhibit low cytotoxicity and are safer. The derivatives can be used for preparing a drug for treating inflammation and inflammation-related diseases such as stroke, neuropathic pain, depression and Alzheimer's disease.
Absstract of: US2025295627A1
A compound nitarsone, or salt thereof is provided for use in the treatment of a neurodegenerative disease, such as Alzheimer's disease (AD), dementia, Parkinson's disease (RD) or amyotrophic lateral sclerosis (ALS). A pharmaceutical composition is also provided that includes compound, or salt thereof for use in the treatment of a neurodegenerative disease.
Absstract of: US2025296939A1
The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keap1 and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION, or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.
Absstract of: EP4620959A1
The present invention discloses cyclic compounds as multi-target kinase inhibitors and preparation methods thereof. The multi-target kinase inhibitors of the present invention are as shown in general formula I, wherein R<sup>1</sup>, R<sup>2</sup>, R<sup>3</sup>, R<sup>3a</sup>, L<sup>1</sup>, L<sup>2</sup>, L<sup>3</sup>, ring A, and ring B are as shown in the Specification and Claims. The present invention also discloses preparation methods of general formula I and its inhibitory activity against multiple kinases. The compounds of general formula I described in the present invention can be used for treating cancers and neurodegenerative diseases such as Parkinson's disease, etc.
Absstract of: EP4620471A2
Pridopidine or a pharmaceutically acceptable salt thereof for use in treating a subject afflicted with amyotrophic lateral sclerosis (ALS).
Absstract of: US2025289803A1
The invention provides a compound of formula (I), or a pharmaceutically acceptable salt, ester or carbamate thereof, or a salt of such an ester or carbamate,wherein either:R1 is OH, and R2 is H; orR1 is H, and R2 is OH.The invention further provides uses of the compounds of formula (I) and compositions comprising compounds of formula (I), including the use of such compounds for the detection of tau deposits, and the use of such compounds and compositions as diagnostic agents in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration, or for the prevention or treatment of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration.
Absstract of: US2025289849A1
The present invention relates to a cell-penetrating peptide and its derivatives to inhibit α-synuclein fibrillation. The present invention specifically relates to a peptide-based inhibitor of Parkinson's Disease. The present invention discloses the identification of the peptides permeable to blood brain barrier for inhibition. α-synuclein fibril formation is observed in the presence of peptides as indicated by SEQ. ID-7, SEQ. ID-8 wherein the SEQ. ID-7, SEQ. ID-8 are truncated versions of SEQ. ID-4 having the homology of 83% and 75% respectively. It provides a peptide having an amino acid sequence of Formula 1. The present invention also provides a pharmaceutical composition comprising a peptide of Formula 1 along with the pharmaceutically acceptable excipient(s) having inhibitory activity against β-sheet polymerisation of amyloidogenic proteins. The analysis showed that peptides corresponding to SEQ. ID-7, SEQ. ID-8 are better inhibitors than SEQ. ID-4 against α-synuclein fibrillation.
Absstract of: AU2024237252A1
Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.
Absstract of: AU2024238598A1
The present invention provides a humanised antibody comprising an antigen-binding domain capable of binding specifically to Aβ42 prefibrillar oligomers with β structure, said antigen-binding domain comprising: (iii) a heavy chain variable region (VH) comprising the sequence of SEQ ID NO.1; or (iv) a light chain variable region (VL) comprising the sequence of SEQ ID NO.2; or a combination thereof. Also provided is the use of the antibody in the treatment of an amyloid disease, and particularly Alzheimer's disease, conjugates and pharmaceutical compositions comprising the antibody, and nucleic acid molecules encoding the antibody or a heavy or light chain polypeptide thereof, as well as vectors and host cells comprising such a molecule.
Absstract of: WO2025194036A1
The present disclosure is generally directed to tetracyclic analogs that modulate autophagy in a subject suffering from alpha-1 antitrypsin deficiency (ATD) and possibly other autophagy associated diseases or disorders, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis.
Nº publicación: WO2025193848A1 18/09/2025
Applicant:
SANGAMO THERAPEUTICS INC [US]
SANGAMO THERAPEUTICS, INC
Absstract of: WO2025193848A1
Provided are compositions for delivery of zinc finger fusion proteins that inhibit expression of tau in the nervous system using the blood-brain barrier penetrant AAV capsid proteins comprising SEQ ID. NO: 1235, and methods of using the compositions to treat neurodegenerative diseases such as Alzheimer's disease, frontotemporal dementia, and other tauopathies.
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