450 results about "Huntingtons chorea" patented technology

Among other things, the present disclosure provides various technologies including chirally controlled oligonucleotide compositions and technologies tor manufacturing and using such oligonucleotide compositions. In some embodiments, the present disclosure provides technologies useful for allele -specific knockdown of mutant Huntingtin transcripts. In some embodiments, tire present disclosure provides technologies useful for reducing the expression, level, amount, and / or activity of mutant Huntingtin transcripts or products thereof. In some embodiments, the present disclosure provides methods for treating Huntington's disease.

The present disclosure provides methods, uses, kits, and compositions comprising a compound of Formula (I), and either a second compound of Formula (I) or a compound of Formula (II). In one aspect, the methods are for treating a neurological disease associated with repeat expansions and / or RAN protein accumulation, reducing the level of one or more repeat associated non-AUG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and / or biological sample. Exemplary diseases associated with repeat expansions include, but are not limited to, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, myotonic dystrophy type 1, myotonic dystrophy type 2, spinocerebellar ataxia, Alzheimer's disease, Huntington's disease, Fragile X Tremor Ataxia Syndrome, and Fragile XE syndrome.

The present invention relates to methods for remotely tuning treatment parameters in movement disorder therapy systems where the subject and clinician are located remotely from each other. The present invention still further provides methods of quantifying movement disorders for the treatment of patients who exhibit symptoms of such movement disorders including, but not limited to, Parkinson's disease and Parkinsonism, Dystonia, Chorea, and Huntington's disease, Ataxia, Tremor and Essential Tremor, Tourette syndrome, stroke, and the like. The present invention yet further relates to methods of remotely tuning a therapy device using objective quantified movement disorder symptom data to determine the therapy setting or parameters to be transmitted and provided to the subject via his or her therapy device. The present invention also provides treatment and tuning remotely, allowing for home monitoring of subjects.

The present disclosure provides compositions and methods useful in the treatment of trinucleotide repeat disorders, including Huntington's disease and Friedreich's ataxia. The present disclosure also provides gRNAs designed to target the HTT or FXN genes. Complexes comprising a base editor and any of the gRNAs disclosed herein are also provided by the present disclosure. The present disclosure further provides polynucleotides, vectors, cells, compositions, and kits. Methods of treating Huntington's disease and Friedreich's ataxia are also provided herein.

The present invention relates to methods for remotely tuning treatment parameters in movement disorder therapy systems where the subject and clinician are located remotely from each other. The present invention still further provides methods of quantifying movement disorders for the treatment of patients who exhibit symptoms of such movement disorders including, but not limited to, Parkinson's disease and Parkinsonism, Dystonia, Chorea, and Huntington's disease, Ataxia, Tremor and Essential Tremor, Tourette syndrome, stroke, and the like. The present invention yet further relates to methods of remotely tuning a therapy device using objective quantified movement disorder symptom data to determine the therapy setting or parameters to be transmitted and provided to the subject via his or her therapy device. The present invention also provides treatment and tuning remotely, allowing for home monitoring of subjects.

The disclosure provides methods for the diagnosis of Huntington's disease. In some embodiments, the method comprises detecting one or more repeat associated non-ATG (RAN) proteins in a biological sample.

The present invention relates to a method of treating a glucose-6-phosphate dehydrogenase dysregulated disorder in a subject in need thereof, the method comprising administering to the subject an effective amount of oleanolic acid, a conjugated salt thereof, or a prodrug thereof. The glucose 6-phosphate dehydrogenase dysregulated disorder can be myofibrillar myopathy, amyotrophic lateral sclerosis, Huntington's disease, Parkinson's disease and Alzheimer's disease caused by BCL2 associated immortal gene mutation.

The present application relates to a sustained release delivery composition of a vesicular monoamine transporter type 2 (VMAT2) inhibitor, a deuterated derivative thereof, a pharmaceutically acceptable salt thereof, an active metabolite thereof, or a prodrug thereof for treatment of hyperkinetic movement disorders including, but not limited to, tardive dyskinesia (TD), Huntington's disease (HD) chorea, tremors, dystonia, chorea, tics, myoclonus, stereotypies, restless legs syndrome, and various other disorders with abnormal involuntary movements. The present application also relates to a sustained release delivery composition of an antipsychotic agent, a pharmaceutically acceptable salt thereof, an active metabolite thereof, or a prodrug thereof for treatment of schizophrenia, bipolar disorder, and other psychiatric diseases or disorders. The method of making or using the composition is also disclosed.

Described herein are crystalline forms of 6-(6-(((1R,2R,3S,5S)-2-fluoro-9-azabicyclo[3.3.1]nonan-3-yl)(methyl)amino)pyridazin-3-yl)-2-methylbenzo[d]oxazol-5-ol (compound A), a small molecule splicing modulator (SMSM) of mRNA, such as pre-mRNA, encoded by genes, for the treatment of Huntington's disease.

The BAG3 proteins are useful in the treatment of Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis, Huntington's disease, Lewy body disease, vascular dementia, mixed dementia and traumatic brain injury, such as chronic traumatic encephalopathy (CTE). A therapeutic BAG3 composition, use of BAG3, and a method of treatment of BAG3 are described.

The invention relates to compounds, compositions, and methods for derepressing RE1 silencing transcription factor (REST) target genes are provided. In particular, a peptide having the sequence TEDLEPPEPPLPKEN (SEQ. ID NO: 1) and EDLEPPEPPLPK (SEQ. ID NO: 15), or the reversed sequences made of D-amino acids (retro inverted, RI) nekplppeppeldet (SEQ ID NO: 16) and kplppeppelde (SEQ ID NO: 17), are disclosed for inhibiting REST activity. The peptides are useful to treat, prevent, or ameliorate conditions such as traumatic brain injury, epilepsy, dementia, Huntington's Disease (HD), chronic pain, brain cancer (including glioblastoma multiforme), pancreatic cancer; diabetes, and peripheral nerve injury.

The disclosure herein relates generally to a method of treating or inhibiting onset of Huntington's disease. This method involves selecting a subject having or at risk of having Huntington's disease and administering to the subject one or modulators of one or more genes as described herein, or proteins encoded therefrom, under conditions effective to treat or inhibit onset of Huntington's disease in the subject.

The present invention includes compositions and methods of treating CNS-related conditions, comprising: administering an effective amount of a compound that lowers the levels of one or more branched-chain amino acids (BCAAs) or metabolites thereof, or any pharmaceutically acceptable salt thereof, wherein the CNS-related conditions is selected from the group consisting of Alzheimer's disease and any related forms of dementia including vascular dementia, Lewy body dementia, frontotemporal dementia, Creutzfeldt-Jacob disease, Wernicke-Korsakoff disease, and Huntington's disease, Multiple sclerosis, Parkinson's disease, autism, Amyotrophic lateral sclerosis (ALS), Hereditary diffuse leukoencephalopathy with spheroids (HDLS) and epilepsy.

To provide 2-aminopyridine derivative compounds for use as inhibitors of nitric oxide synthase.SOLUTION: Disclosed are 2-aminopyridine derivative compounds for use as inhibitors of nitric oxide synthase (NOS). In particular, the field of the present invention relates to 2-aminopyridine derivative compounds for use as inhibitors of neuronal nitric oxide synthase (nNOS), which are formulated as pharmaceutical compositions for treating nNOS-associated diseases and disorders such as Alzheimer's disease, Parkinson's disease, and Huntington's disease, as well as amyotrophic lateral sclerosis, cerebral palsy, stroke / ischemic brain injury, and migraine headaches.SELECTED DRAWING: Figure 1

Aspects of the present disclosure relate to compositions and methods useful for the treatment of nervous system diseases and disorders. In some embodiments, the disclosure provides methods of treating a nervous system disease or disorder comprising administering both a viral vector comprising an interfering nucleic acid (e.g., an artificial miRNA) and a viral vector comprising a CYP46A1 protein. In some embodiments, the disclosure provides methods of treating Huntington's disease comprising administering both a viral vector comprising an interfering nucleic acid (e.g., an artificial miRNA) targeting the Huntington's gene (HTT) and a viral vector comprising a CYP46A1 protein. In some embodiments, the viral vector comprises a modified viral capsid, for example, for preferentially targeting cells in the CNS or PNS.

Described herein are compounds and methods of treating or imaging a disease or disorder, such as Alzheimer's disease, Parkinson's disease, Huntington's disease, ischemic stroke, and prion disease, comprising administering a therapeutically effective amount of a cromolyn ester.

The present invention relates to an improved antibody specifically binding to amyloid-β oligomers (AβOs). Specifically, the present invention relates to an improved form of the antibody W20. Compared with the antibody W20, the improved form of the antibody W20 has a significantly improved affinity to AβOs, and can more significantly inhibit the aggregation of Aβ and the AβOs-induced toxicity of nerve cells, more effectively improve the cognition and memory functions of an Alzheimer's disease model mouse, and reduce pathological changes in the brain of the mouse. The improved form of the antibody can specifically bind to oligomers of an amyloid-β, α-synuclein, mHTT and SOD 1, can inhibit the aggregation and cytotoxicity of various amyloids, and has a better potential for treating various amyloid diseases, such as Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis, than the antibody W20. The improved form of the antibody can specifically bind to a highly toxic amyloid protein oligomer Aβo*3F, and has a better AD diagnosis value. The amino acid sequence of the antibody W20 is as shown in SEQ ID NO: 1.

The use of α-ketoglutaric acid in the preparation of a drug for preventing and treating demyelination-related diseases. The α-ketoglutaric acid or a derivative thereof is used for preventing and / or treating myelin sheath defects in demyelination-related diseases, such as demyelinating diseases, amyotrophic lateral sclerosis, Huntington's disease, hypomyelinating leukodystrophy, Alzheimer's disease, Parkinson's syndrome and diabetes-related visual impairment, and can promote myelin sheath generation, regeneration or repair, and improve the immune microenvironment in the lesion area.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a Huntingtin (HTT) gene, as well as methods of inhibiting expression of an HTT gene and methods of treating subjects having an HTT-associated disease or disorder, e.g., Huntington's disease, using such dsRNAi agents and compositions.

A composition for treating Huntington's disease (HD) by reducing endogenous Huntingtin (HTT) levels in a target includes a siRNA including a siRNA sequence selected from SEQ ID NOs:47 to 149, and a carrier peptide comprising a peptide sequence of SEQ ID NO:1. The carrier peptide may target the nicotinic acetylcholine receptor (nAChR) of neuronal cells in order to deliver the siRNA across the blood-brain barrier (BBB). In some embodiments, the siRNA may be conjugated with the carrier peptide. A method of reducing endogenous HTT levels to treat HD in the target may include administering a composition including a siRNA sequence selected from SEQ ID NOs: 47 to 149 to the target.

The invention relates to neurodegenerative disorders, and particularly to diagnostic and prognostic methods, apparatus and kits for neurodegenerative disorders, such as mild cognitive impairment (MCI), Alzheimer's disease, Parkinson's disease, Huntington's disease or Motor Neurone disease. The invention also extends to methods of detecting novel biomarkers, and methods for treating patients diagnosed or prognosed with a neurodegenerative disorder. A mathematical correlation between the concentrations of Tau protein and T14 peptide is found in samples from healthy subjects without a mood disorder, but not in samples from subjects having neurodegenerative disorders or a predisposition thereto.

The present disclosure provides a compound of Formula (I) or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder, wherein the disease is Huntington's disease ("HD") or spinal muscular atrophy (SMA). This disclosure also features a composition containing the same as well as methods of using and making the same.

Among other tilings, the present disclosure provides various technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing and using such oligonucleotide compositions. In some embodiments, the present disclosure provides technologies useful for allele-specific knockdown of mutant Huntingtin transcripts. In some embodiments, the present disclosure provides technologies usefill for reducing the expression, level, amount, and / or activity of mutant Huntingtin transcripts or products thereof. In some embodiments, the present disclosure provides methods for treating Huntington's disease.

The present invention provides a method of treating neurodegenerative diseases. The method comprises the step of administering to a subject in need thereof an effective amount of a polymer-flavonoid conjugate, or a nanocomplex having an outer shell comprising one or more polymer-flavonoid conjugates and optionally an inner shell comprising one or more flavonoid oligomer and a drug such as anti-CD3 or anti-CD33 encapsulated within the shells. The present method brings therapeutic effective materials through blood-brain barrier to treat neurodegenerative diseases. The present method is effective to treat neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Lewy body dementia and Huntington's disease.

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.

Provided herein are methods for improving working memory and / or executive function in a subject suffering from Huntington's Disease, comprising administering to the subject SAGE-718, an investigational positive allosteric modulator of the NMDA receptor.

Alkoxy-substituted N-benzyl-2-phenylacetamide compounds and derivatives are G-protein coupled receptor (GPR) 88 modulators for use in the treatment of a disease mediated by GPR88. Indications include Tourette's Syndrome, Huntington's Disease (HD), Addiction, Parkinson's Disease (PD), Schizophrenia, and Attention Deficit Hyperactivity Disorder (ADHD), choreiform movements, speech delay, learning disabilities, depression, hyperkinetic movement disorders characterised by chorea and / or dystonia, psychosis, cognitive deficits in schizophrenia, affective disorders, bipolar disorder, Alzheimer's disease and basal ganglia disorders.

Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a huntingtin (HTT) gene. The HTT RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an HTT gene. The HTT 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 HTT RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described HTT RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of HTT gene expression and a reduction in HTT activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including Huntington's Disease.

The present disclosure provides single- or double-stranded interfering RNA molecules (e.g., siRNA) that target a MutS Homolog 3 (MSH3) gene. The interfering RNA molecules may contain specific patterns of nucleoside modifications and internucleoside linkage modifications, as pharmaceutical compositions including the same. The siRNA molecules may be branched siRNA molecules, such as di-branched, tri-branched, or tetra-branched siRNA molecules. The disclosed siRNA molecules may further feature a 5′ phosphorus stabilizing moiety and / or a hydrophobic moiety. Additionally, the disclosure provides methods for delivering the siRNA molecule of the disclosure to the central nervous system of a subject, such as a subject identified as having Huntington's Disease.