29 results about "L-S DISEASE" patented technology

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.

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 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 disclosure provides methods for (i) treating multiple sclerosis in patient, (ii) treating a patient having symptoms of multiple sclerosis, (iii) preventing the onset of multiple sclerosis symptoms in patient having multiple sclerosis or predisposed to multiple sclerosis; (iv) promoting or enhancing immunomodulation and remyelination and / or myelin repair in a subject in need thereof; (v) a neuroinflammatory disorder; (vi) Amyotrophic Lateral Sclerosis; or (vii) a demyelinating disease or disorder or a hypomyelinating condition, each method comprising administering a therapeutically effective amount of a bryostatin compound to the patient.

The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.

The present invention relates to the treatment of a ALS patient with oral fausdil at a dose of 180-240 mg / day, wherein the patient is treated beginning at least 24 months following disease onset. 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.

The present invention relates to the treatment of an ALS patient having disease onset of at least 24 months prior to initiation of treatment with fausdil. Fasudil is administered at a dose of 60-240 mg / day according to specific treatment regimens. 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.

Abstract Dosage regimens for SOD1-targeting antisense oligonucleotides, and salts thereof, are provided. These dosage regimens find use in the treatment of subjects having or at risk of developing amyotrophic lateral sclerosis. Abstract 20 26 20 53 14 06 J ul 2 02 6 A b s t r a c t 2 0 2 6 2 0 5 3 1 4 0 6 J u l 2 0 2 6

Aspects of the disclosure relate to compositions and methods for the diagnosis and / or treatment of C9orf72 negative sporadic amyotrophic lateral sclerosis (C9- sALS). In some embodiments, the disclosure relates to identifying a subject as having C9orf72 negative (C9-) sALS by detecting expression or activity of repeat-associated non-ATG (RAN) translation proteins (e.g., RAN proteins). In some embodiments, the methods and compositions of the disclosure identify certain gene or genes which comprise mutation(s) leading to the expression of the detected RAN proteins, and which were previously unknown to be associated with sALS. In some embodiments, said gene(s) can be used to identify or diagnose subjects having, suspected of having, or at risk of developing sALS which is unrelated to expansion mutations within the C9orf72 and / or SCA36 genetic loci (e.g., C9- sALS). In some embodiments, the disclosure relates to methods of treating C9- sALS by administering to a subject in need thereof an agent that reduces expression or activity of RAN proteins.

The present disclosure features methods and compositions for treating amyotrophic lateral sclerosis (ALS). The disclosed methods comprise administering to a subject having or suspected of having ALS a hematopoietic stem progenitor cell expressing at least one neuroprotective agent. The compositions disclosed comprise hematopoietic stem progenitor cells transduced to express a neuroprotective agent.

Provided is a biomarker for use in the determination or diagnosis of a progression rate of amyotrophic lateral sclerosis (ALS) or a possibility of being affected by ALS and / or the selection or prediction of a therapeutic drug for ALS.　There is discovered a biomarker selected from the group consisting of IL-17A, KLRD1, KRT19, NCF2, TFF2, YTHDF3, Th17, a regulatory T cell (Treg), mature CD8T, naive CD8T, exhausted CD8T, a Classical monocyte, memory CD4T, Th17 / Treg, mature CD8T / naive CD8T, and mature CD8T / exhausted CD8T.

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 are methods for inhibiting the progression of neurodegenerative disease. The methods include administering to a patient suffering from such a disease a composition comprising either deuterated linoleic acid or an ester thereof.

There is provided a method of promoting survival and / or function of an amyotrophic lateral sclerosis (ALS) or ALS-like motor, the method comprising contacting the motor neuron with an agent capable of reducing mitochondrial protein acetylation, particularly an agent selected from a deacetylase activator, such as nicotinamide (NAM) and 7-hydroxy-3-(4′-methoxyphenyl) coumarin (C12), or an acetyltransferase inhibitor, such as GCN5L1 siRNA of SEQ ID NO: 1. Also provided are related agents, oligonucleotides, uses and methods of identifying agents.

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, oligonucleotides comprise various chemical modifications of sugars, nucleobases, and / or internucleotidic linkages and patterns thereof and are usefill for exon skipping. In some embodiments, the present disclosure provides technologies useful for skipping cryptic exons of stathmin-2(STMN2) transcripts. In some embodiments, the present disclosure provides technologies useful for modulating STMN2 transcript splicing. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders or diseases including neurodegenerative diseases, e.g,, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

The present disclosure provides the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in treating a neurological disease associated with repeat expansions and / or RAN protein accumulation, reducing the level of one or more repeat associated non-ATG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and / or biological sample. Also provided is the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in inhibiting RAN protein translation in a subject and in a biological sample (e.g., cells, tissue). Also provided in the present disclosure are pharmaceutical compositions, kits, and uses of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) for treating diseases associated with repeat expansions. Exemplary diseases associated with repeat expansions include, but are not limited to, C9ORFf72 amyotrophic lateral sclerosis (ALS), or C9ORFf72 frontotemporal dementia; myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2); spinocerebellar ataxia; Huntington's disease; Fragile X Tremor Ataxia Syndrome (FXTAS); and Fragile XE syndrome (FRAXE).

A compound of formula (I) or a pharmaceutically acceptable salt thereof is disclosed for use as a medicament. Further, the compound of formula (I) for use in the therapy of amyotrophic lateral sclerosis. Still further, a method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of the compound of formula (I) or a pharmaceutically acceptable salt thereof is disclosed.

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.

The present disclosure provides antibodies that specifically bind to human TDP-43 and methods of using these antibodies to treat patients having TDP-43 associated diseases, including amyotrophic lateral sclerosis (ALS).

This invention discloses a 2,7-disubstituted-5H-pyrrolo[2,3-b]pyrazine derivative, its synthesis method, and its applications, as well as compounds with the structure shown in formula (I), their stereoisomers, or pharmaceutically acceptable salts thereof. These compounds can inhibit MLK3 activity and are used to prepare drugs for the prevention or treatment of diseases caused by MLK3 overexpression, including breast cancer, Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis, ovarian cancer, cervical cancer, prostate cancer, glioblastoma, etc. (I).

The present disclosure relates to a method of treating or ameliorating symptoms of a motor neurone disease and improving motor neuron survival in a subject, more specifically treating or ameliorating symptoms of amyotrophic lateral sclerosis (ALS) and related neurodegenerative disorders. The treatment method comprises administering a Janus kinase (JAK) inhibitor in combination with one or more compounds selected from a glutamate antagonist and an N-methyl-D-aspartate (NMDA) receptor antagonist, in particular baricitinib in combination with riluzole and / or memantine, and compositions and kits thereof for same.

The present application relates to the field of medical systems, in particular to a motion data analysis system and method for amyotrophic lateral sclerosis evaluation, the present application first acquires the disease condition data of each position of the patient and the training data of each position of the selected training motion, and stores them, carries out sclerosis severity analysis and corresponding position motion matching analysis, carries out corresponding position motion safety analysis, then obtains the matching abnormality of motion and patient body through motion matching abnormality and motion safety abnormality analysis, finally selects the training motion corresponding to the smallest matching abnormality as the output of rehabilitation training motion, and the constitution abnormality distribution motion matching abnormality and motion safety abnormality weight is calculated according to the constitution parameter of the patient body and the standard deviation of the safety range, which can accurately customize the rehabilitation training scheme, improve the training pertinence and effectiveness, fully consider the individual difference of the patient, and ensure that the rehabilitation training not only meets the physical condition but also achieves good effect.

The invention discloses a biomarker of amyotrophic lateral sclerosis. On the first aspect, the invention provides application of a substance for detecting a biomarker in preparation of a diagnostic product for amyotrophic lateral sclerosis, and the biomarker comprises at least one of Lyz2, Mup20, Lgals3, Sfn, Apod, Apoe, C1QA, C1QB, SPA3N, CD44 and MT2. According to the scheme, proteomics detection is carried out on medulla oblongata and spinal cord areas of ALS mice and WT mice, a spinal cord and medulla oblongata differential protein network of an ALS pathological model is established, potential biomarkers are screened out, and ALS diagnosis is facilitated through research on the biomarkers.

The present invention relates to antisense oligonucleotides that are complimentary to SOD1, leading to decreased expression of SOD1. Reduced expression of SOD1 is beneficial in medical disorders such as Amyotrophic Lateral Sclerosis.

Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C9ORF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).

The application discloses application of a gamma-aminobutyric acid derivative in treatment of TDP-43 proteinopathy, and discloses application of a gamma-aminobutyric acid derivative or an enantiomer thereof or a pharmaceutically acceptable salt thereof in preparation of a medicine for preventing and / or treating a TDP-43 abnormal aggregation related disease, wherein the TDP-43 abnormal aggregation related disease is amyotrophic lateral sclerosis, sporadic inclusion body myositis, Parkinson's disease, Alzheimer's disease, frontotemporal dementia and Huntington's disease.

The present disclosure provides novel methods for treating or preventing amyotrophic lateral sclerosis (ALS), methods for delaying the onset of neurological symptoms associated with ALS, increasing survival in subjects afflicted with ALS, and attenuating the decline of muscle strength associated with ALS in a subject in need thereof. The present disclosure also provides methods for treating or preventing α-synucleinopathy or TDP-43 proteinopathy. The methods comprise administering to the subject an effective amount of a mitochondriatargeting peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate, and / or solvate thereof.

The present invention relates to an inhibitor of miR-129, relative compounds and pharmaceutical compositions for use in the treatment and / or prevention of amyotrophic lateral sclerosis and Alzheimer's disease. The invention also relates to a method for the diagnosis and / or prognosis of Alzheimer's disease in a subject or to identify a subject at risk to develop amyotrophic lateral sclerosis or Alzheimer's disease and to a method for the measuring the efficacy of a therapy for amyotrophic lateral sclerosis or for Alzheimer's disease and relative kits.