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WO/2021/255420A1 |
There is provided a method of targeting mitochondrial biogenesis and mitochondrial transport from the cell body to axon to protect acutely demyelinated axons from degeneration. The method may comprise increasing mobilization of mitochond...
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WO/2021/257595A1 |
The disclosure provides method of treating muscular dystrophy in a subject in need comprising administering a gene therapy vector, such as adeno-associated virus (AAV) vector, expressing a miniaturized human micro-dystrophin gene in comb...
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WO/2021/256793A1 |
The present invention relates to a composition comprising Lactobacillus bacterium-derived vesicles for preventing or treating a metabolic disease or a muscular disease. As the present inventors identified that when administered to animal...
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WO/2021/254296A1 |
Provided in the present invention are a bioactive substance composition, a serum-free culture medium comprising the composition, and uses thereof. The bioactive substance composition is for use in the serum-free culture medium and/or a c...
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WO/2021/257947A1 |
The present invention provides novel pharmaceutical formulations comprising an antibody that specifically binds to human Activin A. The formulations may contain, in addition to an anti- Activin A antibody, a histidine buffer, an organic ...
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WO/2021/256665A1 |
The present invention relates to a composition which is for preventing, ameliorating, or treating neurological or psychiatric diseases, and comprises, as an active ingredient, vesicles derived from Lactobacillus paracasei. The present in...
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WO/2021/256569A1 |
The present invention provides: compounds that have an inhibitory activity on hematopoietic prostaglandin D synthase (H-PGDS) and are useful for the prevention or treatment of diseases in which the synthase is implicated; and drug compos...
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WO/2021/250541A1 |
Described herein are compounds of Formula I and their pharmaceutically acceptable salts, wherein R1, R2, R3, X1, Y1, Y2, Y3, Y4 and Y5 are defined herein; their use as MC4R antagonists; pharmaceutical compositions containing such compoun...
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WO/2021/251791A1 |
The present invention relates to a trolox-peptide conjugate which exhibits activity that relieves muscle contraction and increases the extracellular matrix, and which can be used as an active ingredient of a drug, a quasi-drug or a cosme...
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WO/2021/252422A1 |
Provided herein are methods for promoting axonal regeneration of sensory neurons and functional recovery of neurons following peripheral nerve injury in a subject experiencing aging-dependent nerve regenerative decline, the method compri...
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WO/2021/252761A2 |
Disclosed herein are monomeric and oligomeric compound embodiments for use as contraceptive agents. Monomeric compound embodiments disclosed herein comprise substituents that facilitate the ability of the compounds to exhibit progestogen...
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WO/2021/251602A1 |
The present invention relates to a function of PHF20 (PHD finger protein 20) in a muscle differentiation mechanism and, more particularly, to a use of a PHF20 gene expression inhibitor or a PHF20 protein activity inhibitor. The PHF20 gen...
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WO/2021/251505A1 |
The present invention addresses the problem of providing an active ingredient capable of improving mitochondrial function. The present invention also addresses the problem of providing a composition for suppressing muscular inflammation ...
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WO/2021/251337A1 |
CB-7 exhibits a weak TLR7 inhibiting effect in normal mice. The present invention provides a novel compound with a stronger TLR7 inhibiting effect than CB-7, a pharmaceutically acceptable salt of said compound, or a prodrug of said compo...
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WO/2021/252314A1 |
The invention provides methods of treating female health conditions related to sex hormones by providing compositions containing the steroid CV-10155.
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WO/2021/249534A1 |
Disclosed are a phthalazinone compound, and a preparation method therefor and the medical use thereof. In particular, disclosed are a compound as represented by formula (I), and a pharmacodynamically acceptable salt, and the use of the c...
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WO/2021/252936A1 |
The present disclosure provides compositions and methods based on the use of 15-PGDH as a therapeutic target in rejuvenation of aging non-skeletal muscle tissues and/or organs. The 15-PGDH inhibitor SW033291 administered intraperitoneall...
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WO/2021/246909A1 |
The present application relates to the field of genetics, gene therapy and molecular biology. More specifically, the present invention relates to an isolated codon-optimized nucleic acid encoding the SMN1 protein (survival motor neuron p...
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WO/2021/246413A1 |
Provided are: an anti-IGF-1 receptor humanized antibody which includes CDRs of a light chain and a heavy chain derived from mice parent antibody IGF11-16, and respective FRs of a light chain and a heavy chain derived from a human antibod...
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WO/2021/246496A1 |
Disclosed herein are methods and compositions for the treatment of age-related dysfunction, particularly, age-related motor impairment.
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WO/2021/246200A1 |
[Problem] To provide a material (an agent) and the like to be used for humans, etc., for suppressing muscle atrophy. [Solution] Provided are: an agent which contains cynaropicrin and which is for suppressing muscle atrophy; and an agent ...
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WO/2021/246779A1 |
The present invention relates to an amide derivative synthesized using probenecid and arginine as reactants, a pharmaceutical composition comprising same for preventing, ameliorating, or treating heart failure, and a method for synthesiz...
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WO/2021/239915A1 |
FoxO inhibitors for use in the treatment of diseases caused by abnormal TDP-43 and/or FUS proteins. The present invention refers to FoxO inhibitors, preferably quinolone derivatives, for use in the treatment of diseases caused by alterat...
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WO/2021/241504A1 |
In one embodiment, the present invention provides an agent for protecting and/or regenerating neuromuscular junctions or an agent for treating and/or preventing a disease in association with neuromuscular junction disorders, each agent c...
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WO/2021/232156A1 |
A pharmaceutical composition comprises: a hydrogel comprising a gel polymer matrix comprising hyaluronan or a derivative thereof and methylcellulose or a derivative thereof; and a cannabinoid, wherein the cannabinoid is dispersed in the ...
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WO/2021/234642A1 |
The present disclosure provides methods of treating ataxia in a subject in need thereof by administering a combination of acetyl-leucine and 4-aminopyridine or a combination of acetyl-leucine and acetazolamide to the subject.
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WO/2021/235293A1 |
Provided is a novel CUG repeat sequence binding agent. This CUG repeat sequence binding agent is configured so as to contain a compound A, the resonance units of which equal 10RU or higher as measured according to surface plasmon reson...
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WO/2021/234562A1 |
A slow-release medical plaster is described, based on diclofenac, specifically a medical plaster based on diclofenac sodium salt, whose formulation allows the release of the active ingredient continuously and at locally therapeutically a...
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WO/2021/233994A1 |
The present invention provides SOCE inhibitors that are useful as therapeutic agents in a variety of applications. The present invention also relates to pharmaceutical compositions, products and kits comprising such SOCE inhibitors, and ...
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WO/2021/231615A1 |
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/2021/231575A1 |
Provided are methods related generally to the fields of molecular biology and virology, in particular the use of immunosuppressive agents and methods for treating using gene therapy, notably Duchenne muscular dystrophy.
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WO/2021/231546A1 |
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/2021/231630A1 |
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/2021/231571A1 |
Provided herein are certain compounds useful as HTT modulators. Such compound are useful in the treatment of Huntington's disease.
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WO/2021/231572A1 |
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/2021/231577A1 |
Recombinant polynucleotides encoding fragments of a human dysferlin protein are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising such recombinant polynucleotides are further ...
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WO/2021/230385A1 |
Polynucleotides comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcriptional activator, and (b) a base sequence encoding a guide RNA targetin...
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WO/2021/231565A1 |
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/2021/227417A1 |
A method for treating spinal muscular atrophy (SMA), comprising the step of administrating a therapeutically effective amount of a plasminogen pathway activator to a subject. Further provided are a pharmaceutical composition containing t...
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WO/2021/231863A1 |
Provided herein is a method for reducing the progression of abnormal muscle pathology and/or reversing abnormal muscle pathology in a patient, wherein the patient has been diagnosed with Pompe disease or is suspected of having Pompe dise...
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WO/2021/229461A1 |
The invention concerns nanovesicles of nanostructured myelin and uses thereof in the treatment of demyelinating and neurodegenerative diseases of the central (CNS) and peripheral (PNS) nervous system. Under another aspect, processes for ...
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WO/2021/224809A1 |
The present invention relates to proline hydroxylase (PHDs) inhibitors acting through pharmacological activation of hypoxia inducible factor-1α (HIF-1α), upregulation of WNT7a expression and iron chelation for use in the skeletal muscl...
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WO/2021/221445A1 |
This specification relates to a compound as a UBR box domain ligand. The present specification provides a small molecule compound that binds to a UBR box domain. In addition, the present specification provides a composition for inhibitin...
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WO/2021/219762A1 |
The invention relates to the use of a recombinant porcine adeno-associated virus (AAV) vector comprising a peptide-modified porcine AAV serotype 1 (AAVpo1) capsid in gene therapy of muscle and/or central nervous system (CNS) disorders, i...
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WO/2021/222327A1 |
Disclosed herein are methods of using probes for high-throughput screening of guide RNA (gRNA) efficiency for Clustered Regularly Interspaced Short Palindromic Repeats (CR!SPR)/CRiSPR-associated (Cas)-based genome editing systems. Furthe...
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WO/2021/218687A1 |
The use of a muscle stem cell in the preparation of a drug for preventing, alleviating and treating metabolic disorders, and a pharmaceutical composition for humans and animals, wherein the pharmaceutical composition comprises the muscle...
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WO/2021/220793A1 |
[Problem] A purpose of the present invention is to provide an autophagy activation agent that can activate autophagy in a living body. [Solution] An extract of sage, fennel, mulberry bark, clove, great yellow gentian, Chinese peony, and/...
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WO/2021/221444A1 |
The present specification relates to a compound as a UBR-box domain ligand. The present specification provides a low-molecular weight compound that binds to a UBR-box domain. In addition, the present specification provides a composition ...
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WO/2021/222165A1 |
The disclosure relates to methods for a genetic and/or metabolic disorder that alters sorbitol metabolism or causes over production of sorbitol, such as SDH deficiency, hereditary neuropathy using aldose reductase inhibitors.
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WO/2021/222268A1 |
Disclosed herein are CRISPR/Cas-based genome editing compositions and methods for treating Duchenne Muscular Dystrophy by restoring dystrophin function. The CRISPR/Cas-based genome editing systems may include a guide RNA (gRNA) targeting...
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