48 results about "Amytrophic lateral sclerosis" patented technology

Modified and stabilized propeptides of Growth Differentiation Factor proteins, such as GDF-8 and Bone Morphogenetic Protein-11, are disclosed. Also disclosed are methods for making and using the modified propeptides to prevent or treat human or animal disorders in which an increase in muscle tissue would be therapeutically beneficial. Such disorders include muscle or neuromuscular disorders (such as amyotrophic lateral sclerosis, muscular dystrophy, muscle atrophy, congestive obstructive pulmonary disease, muscle wasting syndrome, sarcopenia, or cachexia), metabolic diseases or disorders (such as such as type 2 diabetes, noninsulin-dependent diabetes mellitus, hyperglycemia, or obesity), adipose tissue disorders (such as obesity), and bone degenerative diseases (such as osteoporosis).

The invention relates to BCAT inhibitors and the use thereof for treating or preventing neuronal loss associated with stroke, ischemia, CNS trauma, hypoglycemia and surgery, as well as treating neurodegenerative diseases including Alzheimer's disease, amyotrophic lateral sclerosis, Huntington's disease and Down's syndrome, treating or preventing the adverse consequences of the overstimulation of the excitatory amino acids, treating anxiety, psychosis, convulsions, aminoglycoside antibiotics-induced hearing loss, migraine headache, chronic pain, neuropathic pain, Parkinson's disease, diabetic retinopathy, glaucoma, CMV retinitis, urinary incontinence, opioid tolerance or withdrawal, and inducing anesthesia, as well as for enhancing cognition.

The invention provides methods for treating and / or preventing cognitive impairments, dementia, or neurodegenerative diseases and disorders (e.g., Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis) in patients by administering therapeutically effective amounts of an AMPA receptor antagonist (e.g., 1,2-dihydropyridine compounds) and therapeutically effective amounts of nootropics (e.g., cholinesterase inhibitors) to patients. The invention also provides combinations, commercial packages, and pharmaceutical compositions comprising therapeutically effective amounts of AMPA receptor antagonists (e.g., 1,2-dihydropyridine compounds) and therapeutically effective amounts nootropics (e.g., cholinesterase inhibitors). The 1,2-dihydropyridine compound may be, for example, 3-(2-cyanophenyl)-5-(2-pyridyl)-1-phenyl-1,2-dihydropyridin-2-one. The cholinesterase inhibitor may be, for example, donepezil.

The invention relates to BCAT inhibitors and the use thereof for treating or preventing neuronal loss associated with stroke, ischemia, CNS trauma, hypoglycemia and surgery, as well as treating neurodegenerative diseases including Alzheimer's disease, amyotrophic lateral sclerosis, Huntington's disease and Down's syndrome, treating or preventing the adverse consequences of the overstimulation of the excitatory amino acids, treating anxiety, psychosis, convulsions, aminoglycoside antibiotics-induced hearing loss, migraine headache, chronic pain, neuropathic pain, Parkinson's disease, diabetic retinopathy, glaucoma, CMV retinitis, urinary incontinence, opioid tolerance or withdrawal, and inducing anesthesia, as well as for enhancing cognition.

The purpose of the present invention is to provide a novel medicinal agent and a novel method both of which can be used for treating ALS (amyotrophic lateral sclerosis), preventing the progression of the phase of ALS, treating a symptom associated with ALS, or preventing the progression of the phase of the symptom, and both of which can be particularly applied to patients who can be treated by the medicinal agent or the method highly effectively among ALS patients who are needed to be treated. According to the present invention, a medicinal agent which can be used for treating amyotrophic lateral sclerosis, preventing the progression of the phase of amyotrophic lateral sclerosis, treating a symptom associated with amyotrophic lateral sclerosis, or preventing the progression of the phase of the symptom is provided. The medicinal agent comprises 3-methyl-1-phenyl-2-pyrazolin-5-one or a physiologically acceptable salt thereof as an active ingredient, is administered in such a manner that a 14-day dosing period and a 14-day washout period are repeated alternately or in such a manner that a 14-days initial dosing period and a subsequent 14-day initial washout period are set and subsequently a dosing period in which the dosing is carried out for any 10 days of 14 days and a 14-day washout period are repeated alternately, and is administered to a patient who meets specific criteria.

The present invention provides a compound having an MAGL inhibitory action, and expected to be useful as an agent for the prophylaxis or treatment of neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, traumatic brain injury, glaucoma, multiple sclerosis etc.), anxiety disorder, pains (e.g., inflammatory pain, cancerous pain, neurogenic pain etc.), epilepsy, depression and the like.The present invention relates to a compound represented by the formula (I):wherein each symbol is as defined in the description, or a salt thereof.

The present invention relates to the treatment of amyotrophic lateral sclerosis (ALS) and / or the improvement of motor function in individuals in need of such improvement using a melatonin compound or a pharmaceutical salt of the melatonin compound.

The invention relates to an application of an alfuzosin compound in prevention or treatment of Alzheimer's disease and related diseases, and belongs to the technical field of medicines. The alfuzosincompound has the effects of improving mitochondrial metabolism, degrading accumulation of various pathological proteins and improving vascular endothelial cell functions. The invention provides a newpharmaceutical application of the alfuzosin compound. The new pharmaceutical application can provide a new choice for treatment of clinical related diseases. Alfuzosin, an alfuzosin isomers, TZ-md andAZ-md can be beneficially applied to Alzheimer's disease and related complications, such as seeing and hearing impairment, anemia, blood pressure instability, insomnia, depression, limb dysfunction,malnutrition, weakness, dizziness, gait instability and the like, and diseases related to protein accumulation and metabolic disorder, such as Huntington's disease, amyotrophic lateral sclerosis, dementia with Lewis bodies, multi-system atrophy and the like.

The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS). The present invention relates to compositions, methods and processes for the design, preparation, manufacture, use and / or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding at least one small interfering RNA (siRNA) molecules which target the superoxide dismutase 1 (SOD1) gene. Methods for using the AAV particles to inhibit the expression of the SOD1 gene in a subject with a neurodegenerative disease (e.g., amyotrophic lateral sclerosis (ALS)) are also disclosed.

The invention relates to sgRNA and application thereof, and belongs to the technical field of gene editing. The sgRNA sequence damages PTBP1 mRNA in a targeted manner, and the structural domain sequence of the sgRNA sequence is selected from any one of basic sequences shown in SEQ ID NO:1-31, or a truncated sequence which has the sequence length of more than or equal to 20nt and is obtained by truncating at least one basic group at the 3' end and / or the 5' end of the basic sequence, or an extended sequence which has the similarity of more than or equal to 90% with the basic sequence or the truncated sequence. With adoption of the sgRNA, the human PTBP1 mRNA can be destroyed in a targeted manner, the PTBP1 mRNA level can be effectively reduced, and the sgRNA is used for treating PTBP1 mRNAoverexpression related diseases or treating related diseases such as amyotrophic lateral sclerosis, Alzheimer's disease, Parkinson's disease and Huntington's disease by lowering the conventional PTBP1mRNA level, and has important application significance.

This invention relates to a series of N-heterocyclyl hydrazides of Formula I,and pharmaceutical compositions containing them. The compounds of the invention have neurotrophic activity and are useful in the treatment and prevention of neuronal disorders such as Parkinson's disease, Alzheimer's disease, stroke, multiple sclerosis, amyotrophic lateral sclerosis diabetic neuropathy and Bell's palsy.

The invention relates to application of 1-[3-(6, 7-dihydro-1-methyl-7-oxo-3-propyl-1H-pyrazolo [4, 3-d] pyrimidine-5-yl)-4-ethoxy benzenesulfonyl]-cis-3, 5-dimethylpiperazine citrate or Aildenafil citrate in preparation of a medicine for treating neurodegenerative diseases, wherein the treatment of chronic neurodegenerative diseases comprises the treatment of Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis and different types of spinal cerebellar ataxia medicine diseases. The Aildenafil citrate has a wide application prospect in preparation of the medicine for treating the neurodegenerative diseases and treatment of the neurodegenerative diseases.

The present invention relates to O-GlcNAc hydrolase (OGA) inhibitors. The invention is also directed to pharmaceutical compositions comprising such compounds, to processes for preparing such compounds and compositions, and to the use of such compounds and compositions for the prevention and treatment of disorders in which inhibition of OGA is beneficial, such as tauopathies, in particular Alzheimer's disease or progressive supranuclear palsy; and neurodegenerative diseases accompanied by a tau pathology, in particular amyotrophic lateral sclerosis or frontotemporal lobe dementia caused by C9ORF72 mutations.

The invention discloses an application of a c-Abl inhibitor in preparation of a medicine for preventing and / or treating amyotrophic lateral sclerosis. In the application, the invention provides an application of c-Abl protein as a target in screening of drugs for preventing and / or treating amyotrophic lateral sclerosis, an application of SC75741 in preparation of drugs for preventing and / or treating amyotrophic lateral sclerosis, and the like. The c-Abl inhibitor provided by the invention can induce degradation of proteins expressed by amyotrophic lateral sclerosis related pathogenic genes and mutants of the proteins; according to the application, a nervous system model proves that the SC75741 has a remarkable treatment or improvement effect on amyotrophic lateral sclerosis.

The invention provides a biomarker for early diagnosis of sporadic amyotrophic lateral sclerosis and Parkinson's disease and application thereof, relates to the technical field of biomedicine, and solves the technical problem that in the prior art, a biomarker cannot play an effective auxiliary diagnosis role in early clinical diagnosis of sporadic ALS and PD diseases. The biomarker is lncRNAs, and a transcript of the lncRNAs includes one or more of lnc-CNTN4-2: 1, lnc-NR3C2-8: 1, lnc-ABCA12-3: 1, lnc-DYRK2-7: 1 and lnc-POTEM-4: 7. By detecting the expression level of one or more of lnc-CNTN4-2: 1, lnc-NR3C2-8: 1, lnc-ABCA12-3: 1, lnc-DYRK2-7: 1 and lnc-POTEM-4: 7, the detected results can be used as an effective method for early diagnosis of whether a patient has a high risk of suffering from sporadic amyotrophic lateral sclerosis and Parkinson's disease or not, and provides possibility for combined diagnosis of a plurality of biomarkers in the future.

Described herein are methods of treating neuron inflammation conditions, for example, amyotropic lateral sclerosis and prion disease, comprising administering a therapeutically effective amount of a combination of cromolyn or a cromolyn derivative compound and an anti-inflammatory agent.

The invention discloses a gene marker for detecting amyotrophic lateral sclerosis, a detection method and application, and belongs to the technical field of high-throughput sequencing. The detection method comprises the following steps: extracting genome DNA of a detected object, and sequentially carrying out fragmentation, purification and pre-PCR (Polymerase Chain Reaction) amplification reaction on the genome DNA to obtain a target region DNA library; capturing and enriching DNAs of a target gene coding region and an adjacent shearing region of the target region DNA library through the chip to obtain the target region library; carrying out gene detection on the target region library by using a high-throughput sequencing platform, comparing obtained sequencing data with a reference sequence of genome DNA, finding existing gene mutation, and carrying out data analysis to obtain gene mutation information related to amyotrophic lateral sclerosis pathopoiesis. The invention has the advantages of high sensitivity, strong pertinence, comprehensive coverage, large flux, high accuracy and the like, and has huge application space in clinical diagnosis and drug development.

The invention provides a therapeutic agent for amyotrophic lateral sclerosis, comprising: a compound represented by formula (1) (in formula (1), R1 each independently represents an alkyl group containing 1-6 carbon atoms or a 4-hydroxyphenethyl group, and n represents an integer of 1-3); a pharmacologically acceptable salt thereof; or a solvate of the compound and the salt, as an effective ingredient.

Methods of treating, preventing and / or managing central nervous system disorders, such as Parkinson disease, Alzheimer disease, mild cognitive impairment, Huntington disease, Amytophic Lateral Sclerosis, depression and defective long-term memory, and related syndromes are disclosed. Specific methods encompass the administration of a selective cytokine inhibitory drug, or a pharmaceutically acceptable salt, solvate, hydrate, stereoisomer, clathrate, or prodrug thereof, alone or in combination with a second active ingredient. Pharmaceutical compositions, single unit dosage forms, and kits suitable for use in methods of the invention are also disclosed.

The invention relates to BCAT inhibitors and the use thereof for treating or preventing neuronal loss associated with stroke, ischemia, CNS trauma, hypoglycemia and surgery, as well as treating neurodegenerative diseases including Alzheimer's disease, amyotrophic lateral sclerosis, Huntington's disease and Down's syndrome, treating or preventing the adverse consequences of the overstimulation of the excitatory amino acids, treating anxiety, psychosis, convulsions, aminoglycoside antibiotics-induced hearing loss, migraine headache, chronic pain, neuropathic pain, Parkinson's disease, diabetic retinopathy, glaucoma, CMV retinitis, urinary incontinence, opioid tolerance or withdrawal, and inducing anesthesia, as well as for enhancing cognition.

The invention, in general, features a method of treating a neurodegenerative disease (such as amyotrophic lateral sclerosis) or a traumatic brain injury in a subject (e.g., a human) in need thereof, the method comprising administering to the subject a therapeutically effective amount of isolated B cells (such as autologous or allogeneic or xenogeneic B cells).

The present disclosure relates to allogeneic populations of mesenchymal stem / stromal cells and related compositions, which populations and compositions comprise cells pooled from multiple donors, and their use in therapy and / or prevention of inflammatory, autoimmune, transplant related and CNS disorders, in particular CNS such as Amyotrophic Lateral Sclerosis. The present disclosure also relates to methods for obtaining said compositions.

Disclosed are 20-hydroxyecdysone and the derivatives thereof, intended for use in the treatment of a neuromuscular disease such as spinal muscular atrophy or amyotrophic lateral sclerosis, or more particularly in the treatment of a specific disorder of the motor neurons causing alterations in the muscular function occurring in the context of these neuromuscular diseases.

The invention is directed to an in vitro method for performing a differential diagnosis of neurodegenerative diseases in a subject, said subject being selected among subjects suffering from Alzheimer's disease, menial depression, amyotrophic lateral sclerosis, frontotemporal dementia, Parkinson's disease, progressive supranuclear palsy, and / or Parkinson's disease with dementia. Said method comprises the steps of: (a) determining at least five criteria of said subject, (b) comparing said at least five criteria of said subject with reference values by calculating a global note in relation with each neurodegenerative disease, and (c) determining whether said subject suffers from Alzheimer's disease, mental depression, amyotrophic lateral sclerosis, frontotemporal dementia, Parkinson's disease, progressive supranuclear palsy, Parkinson's disease with dementia or mixed dementia.

The present invention relates to novel compounds that inhibit LRRK2 kinase activity, to processes for their preparation, to compositions containing them and to their use in the treatment of or prevention of diseases associated with or characterized by LRRK2 kinase activity, for example Parkinson's disease, Alzheimer's disease and amyotrophic lateral sclerosis (ALS).