54 results about "Motor neurone disease" patented technology

The present invention relates to the use of creatine compound and neuroprotective combinations including creatine, creatine phosphate or analogs of creatine, such as cyclocreatine, for treating diseases of the nervous system. Creatine compounds in combination with neuroprotective agents can be used as therapeutically effective compositions against a variety of diseases of the nervous system such as diabetic and toxic neuropathies, peripheral nervous system diseases, Alzheimer disease, Parkinson's disease, stroke, Huntington's disease, amyotropic lateral sclerosis, motor neuron disease, traumatic nerve injury, multiple sclerosis, dysmyelination and demyelination disorders, and mitochondrial diseases. The creatine compounds which can be used in the present method include (1) creatine, creatine phosphate and analogs of these compounds which can act as substrates or substrate analogs for creatine kinase; (2) bisubstrate inhibitors of creatine kinase comprising covalently linked structural analogs of adenosine triphosphate (ATP) and creatine; (3) creatine analogs which can act as reversible or irreversible inhibitors of creatine kinase; and (4) N-phosphorocreatine analogs bearing non-transferable moieties which mimic the N-phosphoryl group.

There is disclosed herein a composition for treating gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers diarrhea, chronic idiopathic nausea, IBD-associated constipation and diarrhea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinsons disease, MS, Alzheimers Disease, Motor Neurone Disease or autism, the composition comprising: (i) at least two anti-clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (ii) at least one anti-clostridial agent selected from the above combined with an opioid blocking agent. There is also disclosed herein a method of treating various gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers diarrhea, chronic idiopathic nausea, IBD-associated constipation and diarrhea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinsons disease, MS, Alzheimers Disease, Motor Neurone Disease or autism, the method comprising administering orally, via enema or by suppository: (i) a composition of the invention; (ii) at least two anti-clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (iii) at least one anti-clostridial agent selected from the above and an opioid blocking agent to a patient in need of such treatment.

A vaccine for reducing disease progression, and / or protection of motor nerve degeneration, and / or protection from glutamate toxicity in motor neurone disease (MND), particularly amyotrophic lateral sclerosis (ALS), patients, comprising an active agent selected from the group consisting of Cop 1, a Cop 1-related peptide, a Cop 1-related polypeptide, and poly-Glu, Tyr. The active agent is preferably Cop 1 or poly-Glu, Tyr, and can be administered with or without an adjuvant.

The present invention relates to neurological and physiological dysfunction associated with neuron disorders. In (particular, the invention relates to the involvement of vascular endothelial growth factor (VEGF) and homologues in the aetiology of motor neuron disorders. The invention further concerns a novel, mutant transgenic mouse (VEGFm / m) with a homozygous deletion in the hypoxia responsive element (HRE) of the VEGF promoter which alters the hypoxic upregulation of VEGF. These mice suffer severe adult onset muscle weakness due to progressive spinal motor neuron degeneration which is reminiscent of amyotrophic lateral sclerosis (ALS)-a fatal disorder with unknown aetiology. Furthermore, the neuropathy of these mice is not caused by vascular defects, but is due to defective VEGF-mediated survival signals to motor neurons. The present invention relates in particular to the isoform VEGF165 which stimulates survival of motor neurons via binding to neuropilin-1, a receptor known to bind semaphorin-3A which is implicated in axon retraction and neuronal death, and the VEGF Receptor-2. The present invention thus relates to the usage of VEGF, in particular VEGF165, for the treatment of neuron disorders and relates, in addition, to the usage of polymorphisms in the VEGF promotor for diagnosing the latter disorders.

The invention provides a method for determining content of various components in a Chinese medicinal composition freeze-dried injection. The Chinese medicinal freeze-dried injection comprises ginseng and epimedium herb which serve as raw materials and is clinically used for treating amyotrophy and myasthenia gravis which are caused by diseases such as motoneuron diseases (amyotrophic lateral sclerosis, progressive spinal muscular atrophy, progressive bulbar palsy and primary lateral sclerosis), progressive muscular dystrophy, congenital myopathy and the like. In the method, 15 active compounds in the Chinese medicinal freeze-dried injection are qualitatively and quantitatively analyzed simultaneously by a tandem mass spectrometry of a high performance liquid chromatography, and the method can be used for quality control of a product.

The invention provides a method for determining residual organic solvents in the freeze-dried injection of a traditional Chinese medicine composition. The raw materials of the freeze-dried injection of the Chinese medicine include ginseng and epimedium, which are clinically used for motor neuron diseases (amyotrophic lateral sclerosis, amyotrophic lateral sclerosis, spinal muscular atrophy, progressive bulbar palsy, primary lateral sclerosis), progressive muscular dystrophy, congenital myopathy and other diseases caused by muscular atrophy and myasthenia gravis, the method of the present invention uses the top The residual amount of organic solvent can be determined by air chromatography, which can be used for the quality control of the product.

The invention provides a traditional Chinese medicinal composition freeze-drying injection and a preparation method thereof. The traditional Chinese medicament freeze-drying injection is prepared from raw materials comprising ginseng and epimedium herb by extraction and refinement of a modern process and then by a freeze-drying process. The traditional Chinese medicament preparation meets technical requirements of the State Food and Drug Administration (SFDA) on the traditional Chinese medicament injection and is clinically applied to treatment on amyotrophy and myasthenia gravis caused by diseases such as motor neuron diseases (amyotrophic lateral sclerosis, progressive spinal muscular atrophy, progressive bulbar palsy and primary lateral sclerosis), progressive myodystrophy, congenital myopathy and the like. The traditional Chinese medicament freeze-drying injection is administrated clinically through intravenous, intramuscular or acupoint injection.

The present invention relates to therapeutic compounds that are Nrf2-ARE pathway activators suitable for the treatment of diseases known to be mediated by oxidative stress such as motor neurone disease. The invention also includes compounds identified by the methods of the invention for treatment of neurodegenerative diseases.

The present invention relates to therapeutic compounds that are Nrf2-ARE pathway activators suitable for the treatment of diseases known to be mediated by oxidative stress such as motor neurone disease. The invention also includes compounds identified by methods of the invention for treatment of neurogenerative diseases.

The invention provides a method for treating amyotrophic lateral sclerosis (ALS) in a subject. The method comprises administering to the nervous system of the subject a composition comprising a thyroxine protein or a therapeutic fragment or pharmacologic mimic thereof and a pharmaceutically acceptable carrier. The invention also provides a method for treating ALS in a subject that comprises administering to the subject a transthyretin protein, 7B2 protein, a cystatin C protein, a neuroendocrine protein, a cysteine protease inhibitor, or an inhibitor of an enzyme that processes a 7B2 protein. In addition, the invention provides methods for determining the susceptibility of a subject to developing ALS and for determining the progression of ALS in a subject.

An object of the present invention is to provide an agent effective for the treatment and / or prevention of motor neuron disease such as amyotrophic lateral sclerosis (ALS). The present invention provides a therapeutic and / or preventive agent for motor neuron disease comprising the following oligopeptide shown in any of (a) to (c) or a pharmaceutically acceptable salt thereof as an active ingredient: (a) an oligopeptide consisting of the amino acid sequence represented by Ser-Ala-Leu-Leu-Arg-Ser-Ile-Pro-Ala (SEQ ID NO: 1); (b) an oligopeptide consisting of an amino acid sequence having a deletion, substitution, insertion, or addition of one or several amino acids in Ser-Ala-Leu-Leu-Arg-Ser-Ile-Pro-Ala (SEQ ID NO: 1), and having an activity that inhibits neuronal cell death caused by a mutant superoxide dismutase-1 gene; and (c) a modified oligopeptide from the oligopeptide (a) or (b).

The invention provides novel FUS / TLS nucleic acids and proteins that comprise one or more genetic markers (for example, single nucleotide polymorphisms) and methods of use thereof including methods relating to the diagnosis of ALS or other related motor neuron disease by virtue of the presence of the mutant FUS / TLS sequence(s).

Compounds of general formula (I) wherein (I) R1-R4 are each independently selected from H and deuterium; and at least one of R1-R4 is deuterium. The compounds have been found to be particularly useful for treating neurodegenerative conditions and in particular but not exclusively conditions such as motor neurone disease.

Methods for treatment and / or prevention of nerve degeneration in mammals using aza-peptide epoxide caspase inhibitors are provided. Aspects of the present disclosure include aza-peptide epoxide compositions to treat or prevent diseases, for example stroke, Alzheimer's disease, Parkinson's disease, multiple sclerosis, neuropathies, Huntington's disease, dentatorubropallidoluysian atrophy, spinocerebellar atrophies, spinal bulbar muscular atrophy, diabetes, amyotrophic lateral sclerosis and other motor neuron diseases. The disclosed methods can be used in combination with calpain inhibitors to treat disease or pathological conditions related to the activity of caspases and calpain associated with a specific disease or condition. Such treatable conditions include stroke, Alzheimer's disease, Parkinson's disease, multiple sclerosis, neuropathies, Huntington's disease, dentatorubropallidoluysian atrophy, spinocerebellar atrophies, spinal bulbar muscular atrophy, nerve degeneration associated with diabetes, amyotrophic lateral sclerosis and other motor neuron diseases, nerve degeneration secondary to primary demyelinating disorders, among others.

This invention is related to a method of controlling neurodegeneration by increasing CD 147 receptor signaling. Neuroprotection can be achieved suing cyclophilin A or a functional variant, analog or derivative as a ligand for the CDE 147 receptor administered in various means including gene therapy. Conditions treatable with this method include cerebra ischemia, Alzheimer's Disease, Parkinson's Disease, Motor Neurone Disease and / or N=neuronal loss due to trauma and spinal cord damage.

Methods for evaluating subjects having conditions associated with loss of muscle function (e.g., a motor neuron disease, a neuromuscular disease, or a myopathy) by measuring muscle function (e.g., muscle strength) are disclosed.

There is disclosed herein a composition for treating gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers diarrhoea, chronic idiopathic nausea, IBD-associated constipation and diarrhoea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinsons disease, MS, Alzheimers Disease, Motor Neurone Disease or autism, the composition comprising: (i) at least two anti-clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (ii) at least one anti-clostridial agent selected from the above combined with an opioid blocking agent. There is also disclosed herein a method of treating various gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers diarrhoea, chronic idiopathic nausea, IBD-associated constipation and diarrhoea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinsons disease, MS, Alzheimers Disease, Motor Neurone Disease or autism, the method comprising administering orally, via enema or by suppository: (i) a composition of the invention; (ii) at least two anti-clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (iii) at least one anti-clostridial agent selected from the above and an opioid blocking agent to a patient in need of such treatment.

There are provided herein novel monoclonal antibodies that selectively bind and / or activate TrkC receptors, pharmaceutical compositions thereof and the use thereof for treating or preventing conditions which require activation of TrkC, such as amyotrophic lateral sclerosis and other neurodegenerative conditions and motor neuron diseases. The monoclonal antibodies are useful to screen for agents that share the same binding epitope on the TrkC receptor.

The present invention provides compositions and methods for treating ALS and other diseases, particularly motor neuron diseases that are mediated by aberrant aggregation of SOD. Patients with ALS may be treated using a compound of the invention which inhibits SOD aggregation mediated by a Cys-111 residue of SOD, or inhibits SOD aggregation mediated by labile SOD beta-barrel ends. The invention also provides methods for designing compounds capable of inhibiting aggregation.

The present invention provides a novel prodrug of an edaravone compound or a pharmaceutically acceptable salt thereof, a pharmaceutical composition comprising same as an active ingredient, and a use thereof in treatment or alleviation of neurodegenerative and / or motor neuron disease.

The present invention discloses compositions, means and kits thereof for treating neuronal clinical indications in a mammalian subject. The composition comprises, inter alia, a synergistic combination of an anti-inflammatory drug and a DICER activator. The present invention further discloses methods for treating neuronal diseases including Motor neuron diseases (MNDs), ALS, FTD (Frontotemporal Dementia), macular degeneration (AMD) autism, and neurodegenerative diseases such as Parkinson's disease and Alzheimer's disease.

This invention, in at least some embodiments, relates to an inventive molecule, compositions comprising same, and methods of use therof for treatment of a neurological disorder.

There is disclosed herein a composition for treating gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers' diarrhoea, chronic idiopathic nausea, IBD-associated constipation and diarrhoea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinson's disease, MS, Alzheimer's Disease, Motor Neurone Disease or autism, the composition comprising: (i) at least two anti-Clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (ii) at least one anti-Clostridial agent selected from the above combined with an opioid blocking agent. There is also disclosed herein a method of treating various gastrointestinal or neurological disorders, constipation, functional constipation, irritable bowel syndrome, diverticulitis, travelers' diarrhoea, chronic idiopathic nausea, IBD-associated constipation and diarrhoea, pseudo-obstruction, diabetic gastroparesis, cyclic vomiting, reflux oesophagitis, autism enteropathy, flatulence, halitosis, chronic fatigue, bloating, proctalgia fugax, Parkinson's disease, MS, Alzheimer's Disease, Motor Neurone Disease or autism, the method comprising administering orally, via enema or by suppository: (i) a composition of the invention; (ii) at least two anti-Clostridial agents selected from the group consisting of: vancomycin, vancomycin derivatives, a multi-valent polymer of vancomycin, aminoglycosides, nitroimidazoles, ansamysins, nifuroxazide, colchicine, prucalopride, prokinetic agent and 5-aminosalicylic acid; or (iii) at least one anti-Clostridial agent selected from the above and an opioid blocking agent to a patient in need of such treatment.

The invention disclosed herein describes a novel therapeutic target for motoneuron diseases (altered dynamics of microtubules in neurons); methods for measuring the state of activity of this therapeutic target in subjects with established, incipient, or potential motoneuron disease; the discovery of drug agents that modulate neuronal microtubule dynamics in living subjects with motoneuron diseases; the discovery that administration of such agents, alone or in combinations, can improve MT-mediated transport of “synaptic vesicle cargo” molecules along and through axons; the discovery that such modulation of altered microtubule dynamics and improvement in MT-transport of molecules along axons can provide marked neuroprotective therapy for living subjects with motoneuron diseases, including delay in symptoms and prolongation of survival; and the discovery that monitoring of neuronal microtubule dynamics in response to therapeutic interventions in subjects with motoneuron diseases, allows diagnostic monitoring, to optimize therapeutic regimens and treatment strategies in individual subjects or in drug trials. The monitoring involves measuring isotope enrichment in secreted synaptic vesicle cargo molecules.

This invention relates generally to agents and methods for treating the development or progression of a neurodegenerative disease. In particular, the present invention relates to CD14 antagonists for use in treating the development or progression of a neurodegenerative disease, including Motor Neurone Disease (MND) and Dementia disease or associated symptoms. The present invention further provides compositions including such agents.

The present invention provides a novel prodrug of an edaravone compound or a pharmaceutically acceptable salt thereof, a pharmaceutical composition comprising same as an active ingredient, and a use thereof in treatment or alleviation of neurodegenerative and / or motor neuron disease.

The invention discloses medical application of a catalpol and radix astragali extracting solution. According to the medical application disclosed by the invention, an experiment proves that a catalpoland radix astragali water extracting solution or alcohol extracting solution can prevent and control or delay a motor neurone disease and nerve injury; a result shows that by injecting a compositionof catalpol and radix astragali injection liquid, the disease time of amyotrophic lateral sclerosis can be delayed, the survival time is prolonged, an extremity motor function is obviously improved, the catalpol and radix astragali extracting solution has effects of resisting shrinkage on motor neuron cells of spinal cord anterior horn, maintaining or activating a growth state of the motor neuroncells, resisting neuronal apoptosis and protecting, has a function of increasing weight, and also has a good curative effect on a traumatic type nerve injury; moreover, the active ingredients of the catalpol and radix astragali extracting solution disclosed by the invention can be extracted from traditional Chinese medicines, and the used medicines are low in cost, easy to get and good in market prospect.

Disclosed herein are antisense oligonucleotide sequences, and methods for treating neurological diseases. Oligonucleotide inhibitors are described herein. In various embodiments, oligonucleotides target transcripts to treat neurological diseases, including motor neuron diseases and / or neurological diseases. For example, inhibitors of transcripts may be used to treat PD, ALS, FTD, and ALS with FTD.

This invention, in at least some embodiments, relates to an inventive molecule, compositions comprising same, and methods of use thereof for treatment of a neurological disorder.