33 results about "Amytrophic lateral sclerosis" patented technology

The application relates to fused pyrimidinone derivatives of the general formula (I) which act as agonists of Triggering Receptor Expressed on Myeloid cells 2 (TREM2) useful for the treatment of Parkinson's disease, rheumatoid arthritis, Alzheimer's disease, amyotrophic lateral sclerosis, Nasu-Hakola disease, frontotemporal dementia, multiple sclerosis, prion disease, and stroke.

The present invention relates to compositions and methods of GDNF fusion polypeptides, wherein the GDNF fusion polypeptides include an Fc domain, an albumin-binding peptide, a fibronectin domain, or a human serum albumin, joined to a GDNF variant either directly or by the way of a linker. The GDNF fusion polypeptides may used to treat metabolic diseases, such as obesity and Type-1 and Type-2 diabetes, and neurological diseases, such as Amyotrophic lateral sclerosis (ALS) and Parkinson's disease.

The present disclosure relates generally to alkyne containing pharmaceutical agents, and in particular, to phenylethynyl-thiophene based compounds. More particularly, the present disclosure provides a class of compounds that can inhibit and / or attenuate apoptosis via caspase 3 for the treatment of various degenerative disorders. Additionally, the present disclosure relates to methods for treating specific degenerative disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, epilepsy, spinal cord injury, complication due to diabetes, multiple sclerosis (MS), muscular dystrophy (MD), Parkinson's disease (PD), irritable bowel syndrome (IBS) and Alzheimer's disease (AD) in a patient comprising administering to the patient an effective amount of a present compound.

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 present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.

The invention discloses application of bitter gourd exosomes in regulating intestinal flora. The method comprises the following steps: establishing an Alzheimer's disease mouse model, injecting the extracted balsam pear exosome into the mouse body in a gavage manner to serve as an experimental group drug, taking normal saline as a model negative control group, detecting the change of the intestinal flora of the AD mouse by applying a 16S amplicon sequencing principle, and detecting the change of the intestinal flora metabolite of the AD mouse by applying an LS-MS technology. An open field experiment, a nesting experiment and a water maze experiment are adopted to verify the treatment effect of the balsam pear exosome on AD mouse intestinal flora and metabolic level changes thereof. Results prove that the bitter gourd exosome extracted by the invention can effectively improve the intestinal flora micro-ecology of AD mice; the compound can be used for preparing a microecological preparation for regulating intestinal flora of neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, different types of spinal cerebellar ataxia and the like and diseases such as cerebral apoplexy, cerebral injury, epilepsy, tumor and the like.

The present invention addresses the problem of providing an agent for preventing or treating amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), Huntington's disease (HD), spinocerebellar ataxia (SCA), aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging, as well as a more stable antibody that exhibits an effect of preventing or treating these diseases, Alzheimer's disease (AD), or frontotemporal lobar degeneration (FTLD). A human monoclonal antibody that specifically binds to human HMGB1, wherein the human monoclonal antibody (anti-human HMGB1 antibody) comprises a heavy chain CDR1, heavy chain CDR2, and heavy chain CDR3 each consisting of a specific amino acid sequence and a light chain CDR1, light chain CDR2, and light chain CDR3 each consisting of a specific amino acid sequence, is used as an agent for preventing or treating ALS, PD, HD, SCA, aging-related degenerative or neurological disease, brain aging, or diseases associated with brain aging. An antibody in which the light chain complementarity determining region (CDR) 3 of the anti-human HMGB1 antibody has been modified is used.

Disclosed is an application of Teruravone in the preparation of a drug for treating the amyotrophic lateral sclerosis disease. Teruravone can significantly increase the survival rate of TDP-43 M337V stably transfected cells and reduce the LDH leakage rate, and has a potency superior to that of Edaravone, indicating that Teruravone possesses stronger neuroprotective activity. Preliminary clinical trials indicate that after oral administration of Teruravone to an ALS patient for 3 months, the Norris Scale score and the neurofilament light chain protein level in the blood remained generally stable, and the decline in ALSFRS-R score was significantly slowed. Thus, the clinical effect of delaying the disease course of ALS is achieved, and no serious adverse effects were observed.

The present disclosure relates, in some aspects, to compositions of UNC13A antisense oligonucleotides (ASOs) and methods of using ASO to alter UNC13A concealment exon splicing and increase UNC13A protein expression. Such ASO may be used in the treatment of neurodegenerative disorders, in particular neurodegenerative disorders associated with the TAR-DNA binding protein-43 (TDP-43) pathology, such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).

The present disclosure provides cell penetrating agents comprising an intercellular module and an antibody, or antigen-binding antibody fragment thereof, that specifically binds to human TDP-43, and methods of using these cell penetrating agents to treat patients suffering from TDP-43 related diseases, including amyotrophic lateral sclerosis (ALS).

The application relates to fused pyrimidinone derivatives of the general formula (I) which act as agonists of Triggering Receptor Expressed on Myeloid cells 2 (TREM2) useful for the treatment of Parkinson's disease, rheumatoid arthritis, Alzheimer's disease, amyotrophic lateral sclerosis, Nasu-Hakola disease, frontotemporal dementia, multiple sclerosis, prion disease, and stroke.

This disclosure relates to the use of Purine Nucleoside Phosphorylase (PNP) inhibitors such as ulodesine and its salts, in the treatment and / or prevention of diseases associated with nicotinamide adenine dinucleotide (NAD+) depletion, including diseases of mitochondrial dysfunction (including neurodegeneration and peripheral neuropathies), the preservation of cognitive function and in muscular disorders such as sarcopenia; and in metabolic syndrome and associated conditions. In particular the disclosure provides the use of PNP inhibitors such as ulodesine in the treatment of neurodegenerative conditions such as Parkinson's disease and amyotrophic lateral sclerosis.

Methods of treating amyotrophic lateral sclerosis (ALS) are disclosed. Also disclosed is a method of treating C9orf72 amyotrophic lateral sclerosis (C9-ALS).

The present application provides compounds of Formula (I) and related compositions and methods, including methods of therapy for treating nervous system diseases and disorders, including multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS), and methods for in vivo imaging of AMPA receptors or surrogates thereof in the brain. (I)

Pharmaceutical compositions comprising antioxidants as useful therapeutic agents for treatment of amyotrophic lateral sclerosis (ALS), and methods thereof, are disclosed. In particular, the present disclosure provides enteric capsules and intravenous (i.v.) drip of aspirin-mimetic antioxidant drugs in intervention of ALS.

Provided herein are compositions and methods of treating or preventing amyotrophic lateral sclerosis (ALS) in a subject in need thereof, the method comprising administering an agent that modifies neurons by alteration of Kcnn1 protein expression or activity, in turn, neuronal discharge is modified and / or clearance of ALS pathogenic proteins is enhanced or protection against toxicity of the ALS pathogenic proteins is enhanced.

Disclosed herein are UNC13A oligonucleotides having one or more spacer regions or having no spacer regions. In various embodiments, UNC13A oligonucleotides with spacer regions can reduce misspliced UNC13A transcripts and increase full-length UNC13A transcripts, resulting in therapeutic efficacy on nervous system diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

The present invention relates to a composition comprising a c-AMP Response Element-Binding protein 3 (CREB3), in particular a variant of CREB3 protein, or a nucleic acid molecule encoding said CREB3 protein or said variant of CREB3 protein, for use as a medicament, in particular for treating or preventing diseases associated with a degeneration of motor neurons. The present invention further relates to CREB3 protein, in particular said variant of CREB3 protein, as a biomarker for the stratification or the prognosis of patients suffering, or susceptible of suffering, from a disease associated with a degeneration of motor neurons.

The present invention relates to compounds suitable for the imaging of TDP-43 (trans-activation response (TAR) DNA binding protein 43kDa) aggregates. The compounds are useful, for example, in the diagnosis of diseases, disorders or abnormalities associated with TDP-43 aggregates or TDP-43 proteinopathies, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), frontotemporal dementia (FTD) and edge system dominated age-related TDP-43 encephalopathy (LATE). # imgabs0 #

The present application relates to regeneration of functional neurons for the treatment of spinal cord injury and ALS. This document provides methods and materials involved in the treatment of mammals with spinal cord injury (SCI). For example, methods and materials are provided for administering to a mammal suffering from SCI alone a composition containing an exogenous nucleic acid encoding a NeuroD1 polypeptide (or bioactive fragment thereof) or in combination with a Dlx2 polypeptide (or bioactive fragment thereof). This document also provides methods and materials involved in the treatment of mammals suffering from amyotrophic lateral sclerosis (ALS). For example, methods and materials are provided for administering to a mammal suffering from ALS alone a composition containing an exogenous nucleic acid encoding a NeuroD1 polypeptide (or bioactive fragment thereof) or in combination with an Isl1 polypeptide (or bioactive fragment thereof).

Disclosed herein are methods of treating amyotrophic lateral sclerosis in a subject by administering a therapeutic MSC secretory group product prepared by a method comprising culturing a bone marrow-derived MSC under conditions comprising an oxygen tension of less than 5% and a culture medium having a pH of less than 7.

The present invention relates to compounds suitable for the imaging of TDP-43 (trans-activation response (TAR) DNA binding protein 43kDa) aggregates. The compounds are useful, for example, in the diagnosis of diseases, disorders or abnormalities associated with TDP-43 aggregates or TDP-43 proteinopathies, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), frontotemporal dementia (FTD) and edge system dominated age-related TDP-43 encephalopathy (LATE).

The present invention relates to a composition for treatment, and more particularly, to a compound preparation comprising colchicine or a pharmaceutically acceptable salt thereof and metformin or a pharmaceutically acceptable salt thereof as active ingredients, the composition being used for treating, preventing, inhibiting, improving or slowing down neurodegenerative diseases, the invention relates to the treatment of TAR DNA binding protein 43 (TDP-43) aggregate formation, in particular to diseases associated with TAR DNA binding protein 43 (TDP-43) aggregate formation, such as amyotrophic lateral sclerosis, or the occurrence or development of such states. The inventor of the invention confirms that the disease can be effectively treated or prevented by using a compound preparation formed by mixing colchicine and metformin, which is not yet used as the medicine for treating the neurodegenerative disease in the prior art.

The invention discloses an imidazolone compound as well as a pharmaceutical composition and application thereof, the structure of the imidazolone compound is shown as a formula I, and the invention discloses application of the imidazolone compound in preparation of medicines for preventing and / or treating diseases related to MAGL. Comprise depression, anxiety disorder, Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis, multiple sclerosis, neuropathic pain, inflammatory pain, cancerous pain, epilepsy, cancer, alcoholic fatty liver, non-alcoholic fatty liver disease, hepatic fibrosis, cholestasis, nephropathy, sepsis, inflammatory bowel disease and other related diseases. # imgabs0 #

Disclosed herein are UNC13A antisense oligonucleotides with modified backbone structures. The disclosed UNC13A oligonucleotides reduce mis-spliced UNC13A transcripts and increase full length UNC13A transcripts, thereby imparting therapeutic efficacy against neurological diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), or Alzheimer's disease (AD).

This document provides methods and materials involved in treating mammals having a spinal cord injury (SCI). For example, methods and materials for administering a composition containing exogenous nucleic acid encoding a NeuroD1polypeptide (or a biologically active fragment thereof) alone or in combination with a Dlx2 polypeptide (or a biologically active fragment thereof) to a mammal having SCI are provided. This document also provides methods and materials involved in treating mammals having amyotrophic lateral sclerosis (ALS). For example, methods and materials for administering a composition containing exogenous nucleic acid encoding a NeuroD1 polypeptide (or a biologically active fragment thereof) alone or in combination with an Isl1 polypeptide (or a biologically active fragment thereof) to a mammal having ALS are provided.

The present invention provides compounds having the general formula (I) wherein A, B, R1, R2, R3, R5 and R6 are as described herein; compositions comprising the compounds; methods of making the compounds; and said compounds for use in methods of treating or preventing diseases associated with TREM2, such as, for example, Parkinson's disease, rheumatoid arthritis, Alzheimer's disease, amyotrophic lateral sclerosis, Nasu-Hakola disease, frontotemporal dementia, multiple sclerosis, prion disease, and stroke.

The present invention provides compositions and methods for inhibiting the autoimmune component of neurodegenerative diseases and thereby providing a therapeutic effect to patients suffering from such diseases. The compositions and methods comprise immunosuppressive moieties, such as regulatory T cells (Tregs) and proteins expressed by Tregs coupled to a chimeric antigen receptor or that specifically bind to one or more glial cell markers. Therapeutically effective doses of the compounds for treating neurodegenerative diseases, including progressive supranuclear palsy (PSP), Parkinson's disease (PD), Alzheimer's disease, Huntington's disease, amyotrophic lateral sclerosis (ALS), chronic traumatic encephalopathy (CTE), and prion diseases, are disclosed.