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113
resultados
Última actualización
07/08/2025 [09:42:00]
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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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: 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.
Nº publicación: WO2025155903A1 24/07/2025
Solicitante:
TEMPLE UNIV OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION [US]
KORZEKWA KENNETH R [US]
TEMPLE UNIVERSITY-OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION,
KORZEKWA, Kenneth, R
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.
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