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127
resultados
Última actualización
05/07/2025 [08:42:00]
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: 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: 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: 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: US2025213507A1
Tramiprosate and derivatives thereof are provided herein for treating neurodegenerative disorders such as Alzheimer's disease (AD).
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: 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: 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: 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: 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: 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: 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: 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: 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: WO2025136887A1
The present disclosure describes compounds and methods for disrupting the amyloid cascade.
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: 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: 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.
Nº publicación: US2025205368A1 26/06/2025
Solicitante:
NATIONWIDE CHILDRENS HOSPITAL [US]
OHIO STATE INNOVATION FOUND [US]
Nationwide Children's Hospital,
Ohio State Innovation Foundation
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.
BOPI
Sede Electrónica
