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WO/2024/038090A1 |
The instant invention relates to the use of a substance with phosphodiesterase-7 inhibitory activity (PDE7 inhibitor), such as spiro[[1,3]oxazolo[5,4f-]quinazoline-9,1'-cyclohexan)-7-one derivatives, as active ingredient in a therapeutic...
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WO/2024/034644A1 |
The present invention provides a compound for treating or preventing reduced intracellular ATP levels. The present invention relates to a compound represented by formula (I) or a pharmaceutically acceptable salt or solvate thereof, and...
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WO/2024/033467A2 |
The present invention relates to the treatment of myopathy. The inventors report the identification of effective AS-siRNA against the two nucleotide versions of the two non- pathogenic DNM2 SNPs which may be used to silence any mutation ...
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WO/2024/029755A1 |
The present invention relates to an animal model of Charcot-Marie-Tooth disease caused by K141N or K141T mutations in HSPB8. In this animal model expressing the K141N or K141T mutant HSPB8 protein, symptoms of Charcot-Marie-Tooth disease...
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WO/2024/029598A1 |
The present invention provides: a novel method which makes it possible to improve or maintain a muscle amount or muscle strength; and a novel method which makes it possible to prevent obesity, reduce a body fat percentage, reduce a body ...
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WO/2024/029535A1 |
The present invention provides a polypeptide that specifically recognizes a structurally abnormal protein caused by misfolding, etc. in mammals, and a detection agent and a reduction agent for a structurally abnormal protein with the use...
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WO/2024/025418A1 |
The present invention provides methods of increasing Roseburia inulinivorans in a subject for improving muscular strength, muscle mass and/or pulmonary capacity. The invention also provides Roseburia inulinivorans and compositions thereo...
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WO/2024/024565A1 |
The present invention addresses the problem of providing an agent that suppresses cell aging and a drug that prevents or treats diseases and the like caused by cell aging. More specifically, the present invention is a cell aging suppress...
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WO/2024/026470A2 |
Provided, in part, are an anti-human transferrin receptor antigen-binding proteins and fusion proteins comprising an anti-human transferrin receptor antigen-binding proteins (e.g., in scFv, Fab or antibody format) which may be fused to a...
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WO/2024/021383A1 |
The present invention relates to the field of disease treatment. In particular, the present invention provides a gp96 protein and use of a gp96 protein-constructed fusion protein in treating amyotrophic lateral sclerosis. In addition, th...
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WO/2024/022468A1 |
The present invention belongs to the technical field of medicines. Disclosed are use of rhamnose in preparing a medicament for treating or preventing neurodegenerative diseases, a pharmaceutical composition, and use thereof. The present ...
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WO/2024/026224A1 |
The present disclosure relates to methods, uses, compositions, and kits for improving muscle mass, muscle strength, or muscle function or for reducing fatigue, pain, or obesity in a subject using testosterone or testosterone derivative a...
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WO/2024/021876A1 |
Disclosed is use of Eucommia ulmoides male flower extract in the preparation of a food or a medicament for resisting muscle aging, belonging to the technical field of plant extracts. The main active ingredient of the Eucommia ulmoides ma...
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WO/2024/025953A2 |
Disclosed are methods for treating a neuromuscular disorder, muscle disorder, heart disease, pulmonary fibrosis, liver disease, inflammatory bowel disease, or cancer. Also disclosed is a method of enhancing cancer immunotherapy.
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WO/2024/025602A1 |
Compositions and methods for using an Inebilizumab to treat autoimmune disease are disclosed herein. In particular the use of Inebilizumab to treat Neuromyelitis optica spectrum disorder are provided as well as exemplary therapeutic regi...
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WO/2024/024962A1 |
The present invention relates to a therapeutic agent and/or a prophylactic agent for central nervous system diseases associated with abnormal aggregates of brain proteins, the therapeutic agent and/or the prophylactic agent containing, a...
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WO/2024/019080A1 |
The present invention provides: a method for producing umbilical cord-derived mesenchymal cells that highly express a vascular endothelial growth factor, the method comprising a step for culturing an umbilical cord-derived mesenchymal st...
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WO/2024/019533A1 |
The present disclosure relates to a pharmaceutical composition for treating, preventing and/or ameliorating spinal muscular atrophy (SMA) and a method of administration thereof. A pharmaceutical composition for treating, preventing and/o...
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WO/2024/017366A1 |
Provided are a compound of formula (I) having an effect of regulating and controlling the activity of 15-PGDH, a stereoisomer, a tautomer or a form of a mixture thereof, or a pharmaceutically acceptable salt thereof, or a solvate thereof...
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WO/2024/015763A1 |
The present disclosure is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a pregnant mother of a prenatal subject in need thereof, and to pharmaceutical DIPT...
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WO/2024/015329A1 |
The present disclosure provides compositions and methods for preparing autologous satellite cells that have restored a dysfunctional gene involved in muscular dystrophy of a patient. Such implanted satellite cells can promote repair and ...
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WO/2024/011333A1 |
There is provided a method of preventing or treating muscular dystrophy in a subject in need thereof comprising administering a therapeutically effective amount of a stimulator of muscle stem cell (MuSC) function to the subject.
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WO/2024/015762A1 |
The present disclosure is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof, and to administering gene therapy to the subject by intr...
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WO/2024/015453A1 |
Provided are methods for increasing the bioavailability of nucleoside medicinal agents in treating mitochondrial depletion syndromes. In particular, the methods relate to increasing the bioavailability of deoxycytidine and deoxythymidine...
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WO/2024/015765A1 |
The present subject matter is directed to methods of treating Allan-Herndon-Dudley syndrome comprising administering 3,5-diiodothyropropionic acid (DITPA) to a subject in need thereof, wherein the DITPA administration reduces triiodothyr...
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WO/2024/008788A1 |
The invention relates to the use of ebselen or one of the derivatives thereof to treat mitochondrial pathologies or dysfunctions, in particular associated with deficiencies in complex I.
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WO/2024/010030A1 |
The purpose of the present invention is to provide a new prophylactic and/or therapeutic agent that is useful in the prevention and/or treatment of diseases in which symptoms are alleviated or ameliorated by inhibiting myostatin. The p...
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WO/2024/009986A1 |
The present invention provides: an endurance-improving composition that contains, as an active ingredient, triglycerides containing highly unsaturated fatty acids and medium-chain fatty acids as constituent fatty acids; and an endurance-...
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WO/2024/011214A1 |
The disclosure provides a compound of formula I: or a salt thereof, wherein R1, R2, R4, L1, L2, and A have any of the values described in the specification, as well as compositions comprising a compound of formula (I). The compounds 5 ar...
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WO/2024/005564A1 |
The present invention relates to a pharmaceutical composition comprising human pluripotent stem cell-derived mesenchymal stem cells as an active ingredient for the prevention and treatment of rotator cuff syndrome or rotator cuff tears. ...
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WO/2024/005187A1 |
The composition according to the present disclosure includes edaravone and is used to change the expression level of a gene product in a target. The gene product is a gene product from one or more genes selected from KAZALD1, SBK1, SCN2A...
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WO/2024/003798A1 |
The present invention relates to a process for the preparation of 7-(4,7-diazaspiro [2.5] octan- 7-yl)-2-(2,8-dimethylimidazo[l,2-b] pyridazin-6-yl) pyrido-4H-[1,2-a] pyrimidin-4-one represented by the following structural formula (1) by...
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WO/2024/006406A1 |
This disclosure relates to the field of therapeutic tyrosine kinase inhibitors, in particular, Bruton tyrosine kinase ("BTK") inhibitors, for treatment of patients with MG or MS.
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WO/2024/006855A1 |
The present disclosure is directed to methods of treating muscular dystrophy in a subject in need thereof. In certain aspects, the method comprises administering an AAV vector, e.g., an AAVrh74 vector, and an enzyme that cleaves IgG to t...
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WO/2024/003904A1 |
The present invention is directed to combination therapies using compositions of Curcumin and Qing-Dai or extracts of plants producing thereof, and their clinical applications to the treatment of inflammatory conditions in general and to...
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WO/2024/006770A1 |
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating spliceopathy and RNA dominance disorders, including disorders characterized by nuclear retention of RNA transcripts conta...
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WO/2024/003686A1 |
The present invention relates to a composition for topical use comprising p-caryophyllene or oleoresin of Copaifera officinalis containing it, boswellic acids or resin extracts of Boswellia serrata containing it and escin or extracts of ...
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WO/2023/249007A1 |
The present invention provides a calpain inhibitor that has excellent calpain inhibitive capacity. The present invention provides a compound represented by general formula (I), or a salt thereof. (In formula (I), R1 represents a hydrogen...
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WO/2023/249354A1 |
A composition containing demethylzeylasteral or a pharmaceutically acceptable salt thereof, according to the present invention, is highly effective at promoting muscle differentiation and inhibiting muscle degradation, as a glucocorticoi...
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WO/2023/249908A1 |
Snyder-Robinson Syndrome (SRS) is an X-linked intellectual disability disorder caused by a loss-of function mutation in the spermine synthase (SMS) gene. SRS occurs primarily in males. Methods for treating Snyder-Robinson Syndrome or rel...
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WO/2023/247949A1 |
The present invention relates to orodispersible pharmaceutical composition suitable for oral administration comprising Baclofen with at least one disintegrant and at least one diluent. In the preferred embodiment Sodium starch glycolate ...
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WO/2023/246644A1 |
The present invention provides a spinal progenitor cell for treating amyotrophic lateral sclerosis (ALS). The spinal progenitor cell is represented as at least any two of PAX6+, SOX1+, Nkx6.1+, SOX2+, DBX2+, OLIG2+, PAX3+, or Nkx2.2+, an...
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WO/2023/249106A1 |
The purpose of the present invention is to provide a therapeutic agent or a prophylactic agent for amyotrophic lateral sclerosis that has a ferroptosis inhibitory effect. The present invention provides a therapeutic agent or a prophyla...
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WO/2023/250513A1 |
Provided are 13-membered macrolides that can be used to treat proliferative diseases such as cancer as well as genetic diseases that are associated with a premature termination codon mutation or other nonsense mutations. The compounds ca...
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WO/2023/248689A1 |
Provided is a novel climacteric disorder ameliorating agent. A climacteric disorder ameliorating agent according to the present invention includes a powder or an extract of Paecilomyces tenuipes. Such climacteric disorder ameliorating ...
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WO/2023/248008A2 |
Provided herein are degrons, compositions, and chimeric molecules comprising the degrons, and methods of using the degrons and chimeric molecules.
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WO/2023/247764A1 |
The present invention refers to lipopolysaccharides, characterized by comprising: a) O-specific polysaccharide antigen consisting of one or more repeating units, b) core oligosaccharide, and c) lipid A, wherein the lipid A is characteriz...
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WO/2023/249423A1 |
The present invention relates to a composition comprising Lactobacillus gasseri for preventing and treating sarcopenia and, particularly, to: a composition comprising Lactobacillus gasseri BNR17 for preventing, treating or alleviating sa...
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WO/2023/245137A1 |
Provided herein are compounds of Formula (I) and pharmaceutically acceptable salts thereof. Also provided herein is a pharmaceutically acceptable composition comprising a compound of Formula (I), or a stereoisomer or tautomer thereof, or...
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WO/2023/243210A1 |
Provided are a myoblast proliferation promoting agent and a muscular atrophy inhibiting agent that contain a compound represented by structural formula (1).
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