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165
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Última actualización
28/09/2025 [07:28:00]
Resultados 125 a 150 de 165
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: 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: US2025250218A1
Extended chalcone compounds with anti-amylogenic activity were prepared and found to lack cytotoxicity and to promote neuroprotection. Testing on an animal model for Alzheimer's Disease revealed improvements in brain function using the high affinity compounds. The compounds inhibited the aggregation of Aβ42 but not its synthesis. The compounds can be used in new therapies for the prevention and treatment of Alzheimer's disease, including in conjunction with antibodies directed at removing amyloid plaques.
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: 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: 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: 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: 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: KR20250118343A
본 발명은 현호색을 유효성분으로 포함하는 알츠하이머병 예방 또는 치료용 조성물에 관한 것으로, 본 발명의 현호색을 유효성분으로 포함하는 알츠하이머병의 예방 또는 치료용 조성물은 운동 능력 향상 및 인지 기능 개선 효과가 있어 알츠하이머병(AD)의 인지 저하 치료에 효과적으로 사용될 수 있다. 또한, 본 발명의 인실리코 방법을 이용한 치료제 스크리닝 방법은 알츠하이머병(AD)을 치료하는 데 유익한 천연 치료 약초를 판별하는데 유용하게 활용될 수 있는 이점이 있다.
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: CN120051284A
Provided herein are methods of treating early Alzheimer's disease using hydroxypropyl beta-cyclodextrin compositions.
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: 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: 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: CN120417908A
The present invention relates to the compound golesalone for use in the treatment of Parkinson's disease (PD) or for use in the treatment of L-dopa-induced dyskinesia (LID) in patients with Parkinson's disease (PD). Furthermore, the present invention relates to the compound golesalone or a pharmaceutically acceptable salt thereof for use in the treatment of Parkinson's disease (PD) patients, in particular PD patients exhibiting L-dopa-induced dyskinesia (LID).
Resumen de: CN120392785A
The invention discloses novel application of notoginsenoside Rg1, namely application of the notoginsenoside Rg1 in preparation of a cathepsin D (CTSD) inhibitor, the notoginsenoside Rg1 can inhibit CTSD enzyme activity so as to recover lysosome structure and function, relieve endoplasmic reticulum stress reaction and relieve alpha-syn aggregation and dopaminergic neuron apoptosis, has a remarkable protection effect on a Parkinson's disease model, and can be used for preparing a novel anti-inflammatory drug. The invention provides a new thought and a potential drug basis for targeted intervention of Parkinson's disease. The invention discloses a new mechanism that Rg1 plays a role in neuroprotection through a CTSD-lysosome-endoplasmic reticulum stress pathway.
Nº publicación: CN120393055A 01/08/2025
Solicitante:
JINAN UNIV
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Resumen de: CN120393055A
The invention discloses an application of NeuroD1 (NeuroD1) in repairing Alzheimer's disease (Alzheimer's disease). The invention also discloses an application of the carrier for coding NeuroD1 in any one of the following aspects: preventing neuron damage and apoptosis; hippocampus atrophy is inhibited; the neuroinflammation is relieved; repairing blood vessel/blood brain barrier injury; the AD biomarker level of the cerebrospinal fluid is recovered to be normal; the removal of pathological toxic proteins in brain tissues is promoted; glucose metabolism is improved; the space working memory ability is enhanced; and regenerating neurons.
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