301 results about "L-S DISEASE" patented technology

The disclosure provides methods for treating a subject in need thereof comprising administering to the subject a therapeutically-effective dose of psilocybin. The methods described herein may be used to treat a variety of diseases, disorders, and conditions. For example, the methods may be used to treat neurocognitive disorders (e.g., Alzheimer's disease, Parkinson's disease), ADHD, Epilepsy, Autism, Sleep-wake disorders, Chronic pain, Inflammatory Disorders, IBD, Stroke, ALS, and / or Multiple Sclerosis.

The disclosure provides methods for treating a subject in need thereof comprising administering to the subject a therapeutically-effective dose of psilocybin. The methods described herein may be used to treat a variety of diseases, disorders, and conditions. For example, the methods may be used to treat neurocognitive disorders (e.g., Alzheimer's disease, Parkinson's disease), ADHD, Epilepsy, Autism, Sleep-wake disorders, Chronic pain, Inflammatory Disorders, IBD, Stroke, ALS, and / or Multiple Sclerosis.

Disclosed herein are methods and compositions for identifying and / or treating subjects having or likely to have amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). Antibodies specific for one or more di-amino acid repeat-containing proteins are also provided herein.

The invention features polypeptides that include an extracellular ActRIIa variant. In some embodiments, a polypeptide of the invention includes an extracellular ActRIIa variant fused to an Fc domain monomer or moiety. The invention also features pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions involving weakness and atrophy of muscles, e.g., Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, sarcopenia; or cancer cachexia; or metabolic diseases, e.g., obesity, Type-1 diabetes, or Type-2 diabetes.

The present disclosure relates generally to compositions and methods for inhibiting dipeptide repeat protein (DPR)-ribosomal RNA (rRNA) interaction. In particular, the present technology relates to administering a therapeutically effective amount of one or more compositions that inhibit DPR-rRNA interaction to a subject diagnosed with, or at risk for DPR-associated pathologies, e.g., amyotrophic lateral sclerosis or frontotemporal dementia.

In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.

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.

Aspects of the disclosure relate to methods and compositions (e.g., kits) for detecting anti-repeat-associated non-ATG (RAN) protein antibodies in a subject (e.g., a subject that has been administered a therapeutic anti-RAN protein antibody or a vaccine against a disease or disorder associated with RAN protein expression, translation, and / or accumulation, for example amyotrophic lateral sclerosis (ALS) and / or frontotemporal dementia (FTD)). In some embodiments, methods described by the disclosure comprise detecting one or more anti-RAN protein antibodies in a biological sample obtained from a subject by an electrochemiluminescence-based immunoassay using one or more target di-amino acid repeat peptides. In some embodiments, the disclosure relates to kits comprising one or more di-amino acid repeat peptides and an electrochemiluminescence-based immunoassay plate and / or reagents.

Disclosed herein are methods of treating ALS in a subject by administering a therapeutically effective amount of a composition comprising EVs, e.g., derived from neural cells, e.g., neural progenitor cells, to the subject. The EVs can be administered distally or peripherally to the CNS, such that the EVs cross the blood brain barrier and exert their therapeutic function in the CNS. The methods can reduce inflammation (e.g., NLRP3 inflammatory pathway signaling), reduce disease activity or progression, and / or improve motor or neurological performance, signs or symptoms associated with ALS, or survival in the ALS subject as compared to a control ALS subject. The methods of inhibiting necroptosis in a cell by contacting the cell with a therapeutically effective amount of a composition comprising EVs are also provided.

The invention features polypeptides that include an extracellular ActRIIa variant. In some embodiments, a polypeptide of the invention includes an extracellular ActRIIa variant fused to an Fc domain monomer or moiety. The invention also features pharmaceutical compositions and methods of using the polypeptides to treat diseases and conditions involving weakness and atrophy of muscles, e.g., Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, sarcopenia; or cancer cachexia; or metabolic diseases, e.g., obesity, Type-1 diabetes, or Type-2 diabetes.

The invention belongs to the technical field of medicines, and discloses application of hyperforin in preparation of a medicine for preventing or treating amyotrophic lateral sclerosis. The hyperforin can improve the survival rate of stable transfection cell lines of amyotrophic lateral sclerosis related cell models (SOD1G93A / TDP43M337V), and the hyperforin is prompted to play a protective role in the two cell models. After the hyperforin is given, the expression levels of HO-1 and Drp1 proteins in SOD1G93A cells are increased, which indicates that the hyperforin has certain antioxidant activity; and mitochondrial division in cells can be inhibited, so that cell apoptosis is inhibited and retarded, and a protection effect is achieved. Hypericin can help to improve the SOD1G93A cell morphology and promote synaptic generation, and it is prompted that hypericin has a positive effect on maintaining normal functions of cells. In conclusion, the hyperforin plays a protective role in an SOD1G93A cell model and a TDP43M337V cell model, and the mechanism of the hyperforin is possibly related to the antioxidant activity, the mitochondrial function improvement and the like of the hyperforin.

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.

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 invention discloses an application of serratia marcescens in preparation of a medicine for preventing or treating gradual freezing (amyotrophic lateral sclerosis, ALS) or frontotemporal dementia (FTD). Specifically, the serratia marcescens of the present invention can improve the phenotype of an asymptomatic and frontotemporal dementia model S59L by reducing mitochondrial unfolded protein response (UPRmt); meanwhile, expression of excitatory amino acid glutamic acid can be reduced, excitatory neurotoxicity can be relieved, and ALS and FTD phenotypes can be relieved through cooperation of the two.

The present invention relates to activin receptor IIA type variants and methods of use thereof. The present invention features a polypeptide comprising an extracellular ActRIIa variant. In some embodiments, the polypeptides of the present invention comprise an extracellular ActRIIa variant fused to an Fc domain monomer or part. The invention also features the treatment of diseases and conditions involving muscle weakness and atrophy using said polypeptides, such as Duchenne muscular dystrophy, facial shoulder brachial muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, sarcopenia; or cancer cachexia; the present invention relates to pharmaceutical compositions and methods for treating a metabolic disease, such as obesity, type 1 diabetes mellitus or type 2 diabetes mellitus.

Provided herein, inter alia, pharmaceutical formulations for oral administration of ROCK2 inhibitors (e.g., compound A). Also provided are methods for treating a neurodegenerative disease characterized by the degeneration of motor neurons, and particularly amyotrophic lateral sclerosis (ALS), the method comprising administering to the subject in need thereof a therapeutically effective amount of a ROCK2 inhibitor.

Aspects of the disclosure relate to compositions and methods for the diagnosis and / or treatment of certain neurodegenerative diseases, for example those diseases associated with repeat-associated non-ATG (RAN) translation proteins, such as amyotrophic lateral sclerosis (ALS). In some embodiments, the disclosure relates to recombinant adeno-associated viruses (rAAVs) expressing antibodies and antigen-binding fragments thereof that bind to poly(GA) RAN proteins. In some embodiments, the disclosure relates to methods of treating a RAN protein-associated disease by administering to a subject in need thereof the rAAVs.

Provided herein are methods and compositions for the treatment of ALS using chlorite or a pharmaceutical composition comprising sodium chlorite, for a target ALS patient population. Also provided herein are methods for identifying a target ALS patient population likely to be responsive to treatment with chlorite or a pharmaceutical composition comprising sodium chlorite, the methods including identifying ALS patients with elevated plasma C-reactive protein (CRP) levels and / or patients age 40-65, sporadic ALS pathology, or combinations thereof.

The present application relates to siRNA and oligonucleotide agents for use in the prevention or treatment of SOD1-related neurodegenerative diseases or conditions, such as amyotrophic lateral sclerosis, ALS. The oligonucleotide agent comprises a double-stranded targeting oligonucleotide (siRNA) and a non-targeting single-stranded oligonucleotide (ACO), wherein the siRNA targets the mRNA region of the target gene SOD1.

The invention discloses a tomography and targeted energy thermal therapy system and method based on an acousto-optic coupling functional film layer, and relates to the technical field of noninvasive medical diagnosis and treatment. The system comprises an intelligent processing carrier, an acousto-optic coupling function film layer, a scanning mechanism, an energy treatment module and a processing and temperature control module. The intelligent processing carrier at least comprises a 20Hz-20kHz audio unit, a pickup unit, an optical signal unit and a processor; the acousto-optic coupling functional film layer is of a single physical structure and is inseparable, acoustic frequency conversion focusing imaging and targeted warm delivery at 42-47 DEG C are achieved in the same physical area in a time-sharing mode, the acoustic processing layer achieves 1000-8000 frequency doubling through a multi-stage resonance microcavity array, and the echo signal-to-noise ratio is larger than or equal to 15 dB; the scanning mechanism comprises a plurality of modes such as rotation, translation, multi-device array, human body movement, local limb movement, human body bearing support device movement and the like; the energy forms cover all types of light, electromagnetism, radio frequency and the like capable of generating a warm effect, including but not limited to non-contact or contact energy delivery modes. The traditional Chinese medicine composition is suitable for tumors, infections, inflammations, and all lesions sensitive to temperature of 42-47 DEG C including nervous system lesions such as amyotrophic lateral sclerosis, Alzheimer's disease, Parkinson's disease, multiple sclerosis and the like, is low in cost, free of radiation and easy to popularize, and has extremely high creativity and practical value.

The invention discloses an editing system for repairing amyotrophic lateral sclerosis pathogenic mutant genes, and relates to an IS621-bridge-RNA gene editing system capable of being applied to eukaryotic cells, an AAV9 virus vector mediated IS621-bridge-RNA gene editing system and application of the AAV9 virus vector mediated IS621-bridge-RNA gene editing system, and the IS621-bridge-RNA gene editing system, a recombinant expression vector and a gene therapy mode are included.

An object of the present invention is to provide a therapeutic or preventive agent for amyotrophic lateral sclerosis, the agent having inhibitory action against ferroptosis. The present invention provides a therapeutic or preventive agent for amyotrophic lateral sclerosis, the agent containing a tetrahydroquinoline derivative represented by the compound below or a pharmaceutically acceptable salt thereof as an active ingredient.

To provide a method and a composition (e.g., kits) for detecting an anti-repeat-associated non-ATG (RAN) protein antibody in a subject (e.g., a subject being administered therapeutic anti-RAN protein antibodies or vaccines for a disease or disorder related to the expression, translation, and / or accumulation of RAN protein, such as amyotrophic lateral sclerosis (ALS) and / or frontotemporal dementia (FTD)), according to an aspect of the present disclosure.SOLUTION: In some aspects, a method described in the present disclosure includes detecting one or more anti-RAN protein antibodies in a biological sample obtained from a subject by an electrochemiluminescence-based immunoassay using one or more target diamino-repetition-peptides. In some aspects, the present disclosure relates to a kit containing one or more diamino-repetition-peptides and a plate and / or a reagent of an electrochemiluminescence-based immunoassay.SELECTED DRAWING: None

The present invention belongs to the technical field of medicine. Disclosed are a class of magnolol / honokiol derivatives, a preparation method therefor, a pharmaceutical composition thereof and the use thereof. Specifically, disclosed in the present invention are magnolol / honokiol nitrone derivatives as represented by general formulas (I) and (II). Such derivatives are prepared by means of artificial synthesis. Further provided are a pharmaceutical composition containing same, and the use thereof against inflammation, against cerebral infarction, in the treatment of amyotrophic lateral sclerosis, and against cerebral trauma.

To provide stabilization of a retromer for treating Alzheimer disease and other neurodegenerative disorders.SOLUTION: Provided is a method and a composition for increasing and stabilizing a retromer in order to treat and / or prevent Alzheimer disease and other neurodegenerative disorders. Furthermore, provided is treatment of an adenovirus base for treating Alzheimer disease (AD), and other neurodegenerative states, which are Parkinson disease (PD), neuronal ceroid lipofuscinosis (NCL), and transmissible spongiform encephalopathy (TSE or prion disease), multiple system atrophy (MSA), Down syndrome, and hereditary spastic paraplegia for example, and which are progressive supranuclear palsy (PSP), frontotemporal dementia linked to chromosomes 17q21 to 22 and subtype (FTLD-17 / FTLD-Tau) thereof, Lewy body disease (LBD), amyotrophic lateral sclerosis (AES), frontotemporal degeneration (FTD), ALS-FTD and chronic traumatic encephalopathy (CTE) for example.SELECTED DRAWING: None

To provide a C9orf72 oligonucleotide, a composition, and a method for treatment of C9orf72-associated conditions, disorders, or diseases including amyotrophic lateral sclerosis and frontotemporal dementia.SOLUTION: Provided is an oligonucleotide comprising at least one modification of a sugar, a base, or an internucleotidic linkage, wherein the base sequence of the oligonucleotide is or comprises at least 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, or 25 contiguous bases of a base sequence that is at least 80% identical with or complementary to a base sequence of a C9orf72 gene or a transcript thereof, and wherein a nucleobase at a 3' end of the oligonucleotide is optionally replaced by a replacement nucleobase selected from I, A, T, U, G, and C.SELECTED DRAWING: None

Disclosed herein are compounds and methods of use thereof effective for the treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells in the brain and spinal cord, eventually causing loss of muscle strength. The compounds of the disclosure include, cutamesine, a synthetic sigma receptor agonist selective for the 81 receptor, and also a chaperone protein of the central nervous system that plays a key role in the modulation of calcium ions and apoptosis, as well as a sapogenin, such as smilagenin, a non-peptide neurotrophic factor that aids in the reversal of free radical neurotoxicity.

To provide an agent for treating or preventing amyotrophic lateral sclerosis.SOLUTION: This agent for treating or preventing amyotrophic lateral sclerosis essentially comprises at least one active ingredient selected from the group consisting of cycloserine and terizidone, and salts thereof. The active ingredient may be at least one selected from the group consisting of cycloserine and terizidone, and salts thereof. The cycloserine may be D-cycloserine. The cycloserine may be L-cycloserine.SELECTED DRAWING: Figure 8

The present invention relates to methods for the treatment or prevention of symptoms associated with amyotrophic lateral sclerosis (ALS) and related disorders. The present disclosure provides compositions and methods for preventing or treating traumatic brain injuries and other neurological disorders arising from such an injury in a subject by administering to the subject an effective amount of a composition comprising PIF. In certain embodiments, the present disclosure provides compositions and methods for treating traumatic brain injuries and other neurological disorders arising from such an injury in a subject by administering to the subject an effective amount of a composition comprising PIF co-administered with additional agents or treatments.