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163
results.
Updated on
14/10/2025 [07:17:00]
Absstract of: US2025312323A1
This invention relates to methods and materials for treating mammals having, or at risk of developing, one or more movement disorders (e.g., essential tremor, epilepsy, and/or Parkinson's disease). For example, compositions including one or more T-type calcium channel antagonists (e.g., one or more Cav3 antagonists such as CX-8998) are provided, as well as methods for administering such compositions to a mammal having, or at risk of developing, one or more movement disorders (e.g., essential tremor, epilepsy, and/or Parkinson's disease) to treat the mammal.
Absstract of: US2025313574A1
Compound of Formula (I)-(V), compositions comprising at least one compound chosen from compounds of Formula (I)-(V), and methods of using the same, including in treatment of Alzheimer's disease.
Absstract of: AU2024230525A1
The present invention provides for methods of treating obsessive-compulsive disorder (OCD) and OCD-related disorders (body dysmorphic disorder, hoarding disorder, trichotillomania (hair-pulling disorder), excoriation (skin-picking) disorder, substance/medication-induced obsessive-compulsive and related disorder, obsessive- compulsive and related disorder due to another medical condition, and other specified and unspecified obsessive-compulsive and related disorders), Tic disorders including Tourette syndrome, autism spectrum disorder (ASD) and glutamate excitotoxicity related disorders including amyotrophic lateral sclerosis (ALS), Parkinson's disease, traumatic brain injury, multiple sclerosis, Huntington's disease, and schizophrenia, comprising the step of administering an effective amount of a histamine type 1 receptor agonist and/or histamine type 3 receptor antagonist, such as betahistine or its pharmaceutically acceptable salts, analogs, metabolites, prodrugs, derivatives, metabolites, co-crystals, modifications, solvates, hydrates, isotopes, tautomers, esters, polymorphs or stereoisomers.
Absstract of: US2025312408A1
The present invention provides methods for to sex-specific treatment and dose titration of diseases associated with an aberrant functionality of activity-dependent neuroprotective protein (ADNP) and/or cytoskeleton such as progressive supranuclear palsy (PSP), schizophrenia, amnestic mild cognitive impairment (aMCI), Alzheimer's disease, and autism. The treatment in these diseases, e.g., the dose and/or the regimen, differs between sexes and has to be adapted to obtain the desired effect. Specifically, use of davunetide in treatment of women suffering from PSP or of men suffering from schizophrenia or aMCI are provided.
Absstract of: WO2025211909A1
The present invention provides a novel thiophene derivative, an optical isomer thereof, or a pharmaceutically acceptable salt thereof. A novel thiophene derivative according to the present invention has excellent inhibitory activity against KDM4, and thus is useful as a therapeutic agent for brain diseases such as Alzheimer's disease.
Absstract of: WO2025212933A1
The present disclosure provides methods and compositions for the treatment of Tauopathies, including Alzheimer's Disease (AD) and Frontotemporal Dementia and Parkinsonism Linked to Chromosome 17 (FTDP-17), by inhibiting expression of the MAPT gene. Aspects of the disclosure provide engineered inhibitory RNA agents, e.g., siRNA, miRNA, and antisense oligonucleotides, that knock down endogenous mutated or wild-type MAPT transcripts in neuronal cells in a subject. Expression vectors encoding the engineered inhibitory RNA agents, e.g., in an AAV vector, are also provided.
Absstract of: WO2025212467A1
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a microtubule associated protein tau (MAPT) gene. The MAPT RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a MAPT gene. The MAPT RNAi agents are conjugated to an antigen binding protein that may enable subcutaneous delivery of the RNAi agents by facilitating crossing of the blood brain barrier (BBB). Pharmaceutical compositions that include one or more MAPT RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MAPT RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of MAPT gene expression and a reduction in MAPT activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including Alzheimer's disease, Frontotemporal lobar degeneration dementia (FTLD), Progressive supranuclear palsy, and other tauopathies.
Absstract of: US2025312375A1
Disclosed herein are human blood fractions depleted of disease-causing extracellular vesicles, prepared by plasma exchange, that may find use in the treatment of a condition selected from the group consisting of neurodegenerative disease, autoimmune disease, cardiovascular disease, renal disease, and liver disease. In particular, the depleted blood fractions may find use in treating neurodegenerative diseases such as Parkinson's Disease or Alzheimer's Disease.
Absstract of: US2025312385A1
Lachnospiraceae spp and Ruminococcus lactaris new strains of bacteria for use solely or in combination, in the treatment and prevention of memory decline in an individual, in particular declines of aging or Alzheimer's disease-related origin. Compositions, in particular, an oral composition, including the Lachnospiraceae spp and Ruminococcus lactaris strains and uses thereof.
Absstract of: US2025313842A1
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of LRRK2 RNA in a cell or animal, and in certain instances reducing the amount of LRRK2 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 ataxia, neuropathy, and aggregate formation. Such neurodegenerative diseases include Parkinson's disease.
Absstract of: US2025313539A1
Provided in the present invention are an α,β-unsaturated amide compound, and a preparation method therefor, and a pharmaceutical composition and the use thereof. Specifically, provided in the present invention is a compound as represented by formula I, wherein the definition of each group is as described in the description. The compound can be used as a compound for improving cerebral blood flow and is used for preparing a pharmaceutical composition for treating neurodegenerative diseases such as Alzheimer's disease and vascular dementia and strokes.
Absstract of: WO2025212713A1
Methods described herein provide procedures that can be used to rapidly screen drugs for high probability of effectiveness as therapy for amyloid associated neurodegenerative diseases, such as Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), Parkinson's disease, Huntington's disease, and prion-associated diseases by detecting size and/or aggregation of phage incubated with a target compound. The data generated indicate that bacteriophage or phage capsid subunits can switch conformation to a conformation that mimics the neurodegenerative disease-causing conformation of amyloid proteins. This switch has been observed by incubation of bacteriophage T4 with methylene blue, a compound for which literature data indicates has anti-AD activity.
Absstract of: WO2025213066A1
Aspects of the invention provide an AAV product that delivers an GBA1 gene supplementation strategy throughout the human CNS at levels of DNA Biodistribution, RNA expression, and GBA1 protein levels expected to restore function in patients with Parkinson's disease.
Absstract of: WO2025210087A1
The present invention relates to compounds which are suitable for imaging TDP-43 (Transactive response (TAR) DNA binding protein 43 kDa) aggregates. The compounds can be used, for example, for diagnosing a disease, disorder or abnormality associated with TDP-43 aggregates or a TDP-43 proteinopathy, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE).
Absstract of: WO2025210088A1
The present invention relates to compounds which are suitable for imaging TDP-43 (Transactive response (TAR) DNA binding protein 43 kDa) aggregates. The compounds can be used, for example, for diagnosing a disease, disorder or abnormality associated with TDP-43 aggregates or a TDP-43 proteinopathy, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE).
Absstract of: US2025313570A1
The present invention relates to a use of a novel compound, for preventing, improving or treating amyotrophic lateral sclerosis (ALS), wherein the present inventors have found that SOD1 aggregation is one of the important causes of ALS, and have proposed the possibility that WT-SOD1 aggregation, caused by suppressing the regulation of intracellular stress or TDP-43, may be a cause of sALS. In addition, the present inventors have discovered the novel compound PRG-A-01(SLC-B036) as a SOD1 aggregation and misfolding inhibitor.
Absstract of: MX2025006287A
The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.
Absstract of: WO2025201511A1
The present invention relates to an indazole derivative and a preparation method therefor and a use thereof. The indazole derivative can be used for LRRK2 kinase inhibition or for treating Parkinson's disease (PD).
Absstract of: US2025302843A1
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.
Absstract of: WO2025207476A1
A method of treating amyotrophic lateral sclerosis (ALS), includes: determining for a patient diagnosed with ALS, a pre-treatment amount of neurofilament light chain (NfL) in the patient's serum or plasma; orally administering to the patient a first rho kinase inhibitor in a predetermined amount for a predetermined period of time; determining a post-treatment amount of NfL in the patient's serum or plasma; when the post-treatment of amount of NfL is determined to be lower than the pre-treatment amount of NfL, orally administering to the patient a second rho kinase inhibitor in a therapeutically effective amount for treating ALS; and when the post-treatment of amount of NfL is determined to be not lower than the pre-treatment amount of NfL, withholding administration of the second rho kinase inhibitor to the patient.
Absstract of: WO2025201405A1
A pharmaceutical composition comprising at least three components selected from the group consisting of Compound A, Compound B, Compound C, and Compound D, and their use in treating a neurodegenerative disease (e.g., Alzheimer's disease) and improving cognition or memory.
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: 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: 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.
Nº publicación: US2025304970A1 02/10/2025
Applicant:
IONIS PHARMACEUTICALS INC [US]
Ionis Pharmaceuticals, Inc
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.
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