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WO/2023/091606A1 |
Substituted pyridazinone compounds, conjugates, and pharmaceutical compositions for use in the treatment of neuromuscular diseases, such as Duchenne Muscular Dystrophy (DMD), are disclosed herein. The disclosed compounds are useful, amon...
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WO/2023/088455A1 |
Provided are a siRNA for inhibiting expression of a complement component 3 (CC3) gene, a pharmaceutical composition containing the siRNA, and a siRNA conjugate. Each nucleotide in the siRNA is independently a modified or unmodified nucle...
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WO/2023/089313A1 |
There is described a compound for use in the treatment of a mitochondrial DNA disorder, wherein the compound is a glycolysis inhibitor, an inhibitor of glutamine consumption or is L-asparaginase or pegaspargase. The glycolysis inhibitor ...
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WO/2023/090662A1 |
In the present invention, calsequestrin 1 (CASQ1) binds to STIM2 through the C1 (N-terminal 92 amino acid sequence) site of CASQ1 and the expression of CASQ1 or the C1 site thereof (CASQ1-STIM2 binding) is identified to lower store-opera...
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WO/2023/087061A1 |
An isolated or purified antisense oligomer which has a modified backbone structure for modifying pre-mRNA splicing in the PPIF gene transcript or part thereof.
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WO/2023/090411A1 |
Provided is a pharmaceutical composition for treating and/or preventing at least one among heart disease, heart disease complications, skeletal muscle disease, and skeletal muscle disease conditions, the pharmaceutical composition having...
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WO/2023/090409A1 |
In one embodiment, the present invention pertains to a peptide having affinity for the human transferrin receptor, and to applications of the same, wherein in order to pass through the blood-brain barrier, the peptide can be used by bind...
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WO/2023/091930A1 |
The present disclosure provides methods of treating or reducing the symptoms of muscular dystrophy in a human patient between the age of 1 day and 18 years old, comprising administering to the human patient in need thereof a therapeutica...
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WO/2023/083718A1 |
The invention relates to at least one phytoecdysone and/or at least one semi-synthetic derivative of 20-hydroxyecdysone, and at least one active ingredient having the ability to increase the production of functional SMN protein, for use ...
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WO/2023/085820A2 |
The present invention relates to a pharmaceutical composition for preventing or treating muscle diseases. Particularly, an elderberry extract or a monosaccharide- and amino acid-coupled compound, according to the present invention exhibi...
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WO/2023/086988A1 |
The current disclosure provides novel methods and compositions to suppress immunity and are useful for treating autoimmune and inflammatory conditions, and in some circumstances, cancers. Accordingly, aspects of the disclosure relate to ...
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WO/2023/086601A1 |
The invention provides novel pharmaceutical compositions and methods thereof for therapeutic use in treating various congenital diseases and conditions, e.g., Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental De...
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WO/2023/086864A1 |
Aspects of the disclosure relate to oligonucleotides designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of dise...
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WO/2023/085402A1 |
Provided is a novel peptide molecule having an inhibitory activity on the formation of leukocyte extracellular traps. In the description, a peptide molecule having a structure containing an amino acid sequence represented by formula (1) ...
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WO/2023/086854A1 |
This disclosure relates to the unexpected discovery that agents capable of inhibiting calcium channels, such as calcium channel blockers, are effective in treating or alleviating the symptoms of myotonic disorders. In patients who have b...
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WO/2023/079079A1 |
The disclosure relates to methods of preserving myelination of axons in a human subject having a demyelinating disease by administration of an effective amount of a monoselective S1P receptor modulator, such as ponesimod.
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WO/2023/078807A1 |
The present invention relates to a pharmaceutical composition comprising a class Ila HDAC inhibitor characterised by a trifluoromethyloxadiazole group and/or 5-Aza-2-deoxycytidine for use in treating myopathy.
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WO/2023/081157A1 |
A method including administering a therapeutically effective amount of a therapeutic agent to a subject for improving exercise performance, treating obesity, fatty liver, rhabdomyolysis, Leigh syndrome, mitochondrial Complex I deficiency...
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WO/2023/079430A1 |
The invention provides antibodies, and antigen-binding fragments thereof, that specifically bind to GDF15, as well as methods and uses for the antibodies.
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WO/2023/073134A2 |
The present invention concern endothelin receptor antagonists for use in the treatment or the prevention of muscle fibrosis.
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WO/2023/076986A1 |
The present disclosure provides compositions and methods based on the use of 15-PGDH and/or PGE2 as a therapeutic target, for example, to regenerate or rejuvenate injured, damaged, aged, or diseased tissue and/or injured, damaged, aged, ...
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WO/2023/074937A1 |
The present description pertains to a novel compound as a ligand binding to a UBR box domain that is involved in an intracellular protein degradation pathway, and a use thereof.
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WO/2023/077120A1 |
Aspects of the disclosure relate to compositions comprising a plurality of complexes comprising an antibody (e.g., anti-TfRl antibody) covalently linked to one or more oligonucleotides (e.g. a phosphorodiamidate morpholino oligomer), eac...
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WO/2023/075491A1 |
The present invention relates to a composition comprising a peony extract for preventing, alleviating, or treating muscular dystrophy. The composition comprising a peony extract can be used to reduce muscle damage and HMGB1 levels, inhib...
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WO/2023/070156A1 |
The present invention relates to productive tissue repair and regeneration, and in particular poylpeptides, compositions including said polypeptides, and methods of using said polypeptides or compositions for productive tissue repair and...
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WO/2023/070087A1 |
The present disclosure relates generally to methods of preventing, reducing risk of developing, or treating Duchenne muscular dystrophy, Becker muscular dystrophy, Limb- Girdle Muscular Dystrophies (LGMD), Collagen Type VI-Related Disord...
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WO/2023/067127A1 |
The present invention relates to the use of shRNA inhibiting ORAI1 expression for the treatment of a disease selected from the group consisting of tubular aggregate myopathy (TAM) and Stormorken syndrome (STRMK).
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WO/2023/068835A1 |
The present disclosure relates to a novel PAK4 inhibitor and uses thereof. In addition, the present disclosure newly reveals the role of PAK4 in the process of tissue damage caused by ischemia-reperfusion or hypoxia-reoxygenation, and pr...
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WO/2023/066831A1 |
The present invention relates to an amino acid composition comprising threonine, serine, proline, leucine, and cysteine for use in improving liver function in a subject. The present invention also relates to an enteral nutritional compos...
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WO/2023/066832A1 |
The present invention relates to an amino acid composition comprising threonine, serine, proline, leucine, and cysteine for use in preventing and/or reducing glutamine deficiencyand/or arginine deficiency in a subject. The present invent...
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WO/2023/068209A1 |
A compound represented by formula (I) is used as an active ingredient of an AMPK activator, a motor function improving agent, a muscular endurance improving agent and a muscle atrophy preventing agent according to the present invention. ...
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WO/2023/068747A1 |
The present invention relates to a composition comprising reboxetine as an active ingredient for prevention or treatment of sarcopenia. In the present invention, reboxetine is identified to have an effect of alleviating the atrophy of my...
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WO/2023/068366A1 |
The present invention addresses mainly the problem of providing a novel composition or pharmaceutical that targets the mitochondrial network etc. and exhibits excellent anticancer activity while having a minimal effect on normal cells.ã€...
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WO/2023/063033A1 |
[Problem] To provide a novel sarcopenic obesity inhibiting composition, and a prophylactic and/or therapeutic composition containing the same for improving sarcopenic obesity induced by diabetes. [Solution] A sarcopenic obesity inhibitin...
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WO/2023/062511A1 |
The disclosure relates to methods of treating multiple sclerosis. Also provided are pharmaceutical products containing ponesimod, instructions for use of ponesimod, and methods for reducing clinical management events before or during tre...
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WO/2023/061935A1 |
The disclosure relates to methods of treating multiple sclerosis. In certain aspects, methods of reducing corticosteroid use in a patient with multiple sclerosis are disclosed.
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WO/2023/061939A1 |
The disclosure relates to methods of treating multiple sclerosis. In certain aspects, methods of treating early-stage multiple sclerosis in a patient are disclosed.
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WO/2023/061918A1 |
The disclosure relates to methods of treating multiple sclerosis and maintaining or maximizing vaccine effectiveness. In certain aspects, the methods comprise administrating ponesimod, administering a vaccine, and interrupting the admini...
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WO/2023/061488A1 |
Provided herein is a traditional Chinese medicine, Mu Dan Pi (Moutan radicis cort), that has the potential to be used for the prevention or treatment of cachexia and muscle loss in cancer patients. The composition is a novel therapeutic ...
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WO/2023/061499A1 |
Provided are codon-optimized sequences coding for SMN polypeptide and recombinant adeno-associated virus (rAAV) vectors comprising one of said sequences under the control of a hybrid promoter. Also provided are viral particles comprising...
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WO/2023/064947A1 |
Provided herein are antibodies that specifically bind to a peptide antigen. Also provided are methods of detecting, isolating or quantifying the peptide antigen. In some embodiments, the peptide antigen is released from dystrophin or mic...
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WO/2023/060269A1 |
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to be a tropic or have limited tropism which may be further engineered by insertion of a targeting domain s that confer and/o...
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WO/2023/057404A1 |
The invention relates to 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2
-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one, also known as risdiplam, for use in the treatment of spinal muscular atrophy (SMA) with GYM329, its pha...
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WO/2023/060272A2 |
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences and/or amino acid substitutions that confer and/or enhance desired properties, particularly i...
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WO/2023/056897A1 |
Provided is the use of a c-Abl inhibitor in the preparation of a drug for preventing and/or treating amyotrophic lateralizing sclerosis. Provided are the use of a c-Abl protein as a target in screening a drug for preventing and/or treati...
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WO/2023/059590A1 |
The present disclosure relates to compositions and methods for treaty obesity-related diseases. In some aspects, methods are provided for increasing the expression and/or activity of Dj 1 protein in a subject in need thereof.
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WO/2023/060097A1 |
Provided herein are methods and compositions for the treatment of ALS using chlorite or a pharmaceutical composition comprising sodium chlorite, for a target ALS patient population. Also provided herein are methods for identifying a targ...
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WO/2023/055774A1 |
Provided herein are oligonucleotides, peptide-oligonucleotide-conjugates, and a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of human acid alpha-glucosidase (GAA) gene having at least one purine and p...
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WO/2023/054625A1 |
A problem addressed is to provide a useful means for increasing muscle mass. A composition containing threonic acid, one or more bacteria selected from Bifidobacterium spp., Lactobacillus spp., Lacticaseibacillus spp., Lactiplantibacillu...
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WO/2023/054626A1 |
Provided is a cyclopropanamide derivative. A compound represented by formula (1) [wherein: n represents an integer of 0, 1, 2 or 3; ring A represents (1) 4-pyridyl which may be substituted with 1 to 4 substituents, or (2) phenyl which is...
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