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WO/2019/115781A1 |
The present invention relates to compounds suitable for treating, ameliorating and/or preventing neuromuscular disorders, including the reversal of drug-induced neuromuscular blockade. The compounds as defined herein preferably inhibit t...
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WO/2019/115791A1 |
The present invention relates to anti-CD45RC antibodies, for use in the treatment of monogenic diseases caused by genes not associated with immune function but whose deficiency is associated with inflammation and/or immune reactions (suc...
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WO/2019/115711A1 |
The present invention relates to compounds for the treatment of diseases related to DUX4 expression, such as muscular dystrophies. It also relates to use of such compounds, or to methods of use of such compounds.
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WO/2019/117148A1 |
The purpose of the present invention is to provide a novel low molecular-weight compound having orexin receptor agonist activity that is expected to be useful for the prevention or treatment of narcolepsy, etc. The present invention prov...
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WO/2019/107532A1 |
The present invention provides an aptamer that binds to chymase and includes a consensus sequence that is represented by UAACR1N1R2GGGG (provided that the uracil may be thymine). (In the formula, R1 and R2 are arbitrary single bases, and...
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WO/2019/107057A1 |
[Problem] The purpose of the present invention is to provide a peptide, etc., that is capable of suppressing muscle atrophy. [Solution] A peptide comprising an amino acid sequence represented by any of SEQ ID NOS: 1-13. A composition hav...
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WO/2019/101700A1 |
A composition comprising at least one of oleuropein or curcumin can treat or prevent sarcopenia, reduce a loss of at least one of muscle quality or muscle mass, increase at least one of muscle quality or muscle mass, and/or improve recov...
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WO/2019/103926A1 |
The present disclosure provides methods of treating a patient comprising administering a p38 inhibitor for the treatment of FSHD. In some embodiments, the present methods comprise using one or more p38 inhibitors as a therapeutic agent f...
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WO/2019/101709A1 |
Are disclosed oxadiazole derivatives, their use as medicaments and in particular for the treatment of diseases associated with the presence of a nonsense mutation in the gene or a premature stop codon in the mRNA, pharmaceutical formulat...
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WO/2019/099711A1 |
The disclosure is directed to the use of a pharmaceutical product to accelerate recovery of adverse side-effects resulting from neurotoxin therapy for bladder dysfunction.
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WO/2019/095064A1 |
The present description relates to the use of a SRSF3 agent for regulating the function of a myeloid cell, such as a microglial cell and/or monocyte, for treating neurological conditions, cancers, bacterial infections and viral infection...
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WO/2019/099800A1 |
Trophic factor expressing monocyte cells derived from gene-edited induced pluripotent stem cells, methods for making and using.
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WO/2019/099823A1 |
Methods and mechanisms for treating and/or alleviating myotonic dystrophy type 1 (DM1 by editing the DMPK gene. Editing of the DMPK gene may take place in vivo, or may involve ex vivo correction followed by implantation of genome-correct...
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WO/2019/100053A1 |
Compositions and methods for modulating HIF-2α to meditate of hypoxia signaling in satellite cells and applications thereof for improving skeletal muscle generation and repair are provided. For example, methods of enhancing, increasing,...
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WO/2019/092251A1 |
The present disclosure relates to a BIN1 protein or a BIN1 nucleic acid sequence producing or encoding the same, for a use in the treatment of X-linked centronuclear myopathy. The present invention provides compositions and methods for t...
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WO/2018/225065A9 |
Novel aminoglycosides, represented by Formulae (e.g., a compound of Formula A, B, I, I*, III or III*, including compounds represented by Formula Ia, I**, I*a, I*b, IIIa, III**, III*a and, III*b), as defined in the instant specification, ...
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WO/2019/086407A1 |
The invention relates to the field of protein-dense liquid nutritional compositions for use in the treatment and/or prevention of a condition linked to loss of muscle mass and/or strength. Provided is a heat-treated liquid high-protein c...
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WO/2019/090085A1 |
Provided herein are compounds of formula (I), compositions, and methods useful for modulating the integrated stress response (ISR) and for treating related diseases, disorders and conditions. (Formula I).
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WO/2019/088159A1 |
The present application provides a substituted purine compound represented by formula (1) and a pharmaceutically acceptable salt thereof, which exhibit a TLR7-inhibiting effect and are useful for therapy, etc., of autoimmune diseases. [I...
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WO/2019/087280A1 |
The present invention provides a muscle-building composition or the like that has few side effects, is very safe, and enables efficient buildup of muscles. Disclosed are: a muscle-building composition that contains, as an active ingredie...
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WO/2019/085872A1 |
Disclosed are methods and compositions useful in preventing or treating a disease related to muscle wasting by using acidic fibroblast growth factor (aFGF).
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WO/2019/088010A1 |
The present invention provides a testosterone-containing transdermally administrable preparation having excellent testosterone cutaneous permeability and excellent physical properties. More specifically, the present invention provides a ...
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WO/2019/090078A1 |
Provided herein are compounds, compositions, and methods useful for modulating the integrated stress response (ISR) and for treating related diseases; disorders and conditions.
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WO/2019/090171A1 |
Provided herein are methods and compositions for increasing muscle strength, and for treating muscle wasting disorders, muscle degenerative disease, or exercise-induced weakness, and cancer.
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WO/2019/084499A1 |
Disclosed herein are methods and compositions for the treatment of facioscapulohumeral muscular dystrophy. In some cases, the methods and compositions involve the use of kinase inhibitors including Src, Syk, Abl, Tie, Flt, ErbB, Trk, PRK...
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WO/2019/082940A1 |
The purpose of the present invention is to provide a medicament containing as an active ingredient a compound having ryanodine inhibitory activity, in particular, RyR1 inhibitory activity. Provided is an agent for treating or preventing ...
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WO/2019/077810A1 |
Provided is an agent or composition useful for the proliferation of myocytes, etc. The agent or composition comprises component (A) which is at least one member selected from acteoside, echinacoside and salts thereof and component (B) wh...
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WO/2019/077149A1 |
The invention relates to a pharmaceutical composition for targeting drug delivery including gene delivery to regenerating muscle tissue, comprising at least a therapeutic drug or gene, associated to a syncytin protein, and its use in the...
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WO/2019/079596A1 |
The present disclosure relates to methods and compositions for treating immune-mediated diseases. In some aspects, the disclosure relates to methods for treating immune-mediated diseases by administering an EHMT2 inhibitor in combination...
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WO/2019/077258A1 |
The present invention relates to the field of oil-in-water emulsions and more particularly to oil-in-water emulsions containing essential oils and formulated without a surfactant or any other substitute substance.
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WO/2019/078711A1 |
The present invention relates to the use of an agent that inhibits POH1 to treat a patient suffering from or at risk of developing muscle degeneration. The invention arises from the discovery that POH1 regulates PABPN1 protein and thus o...
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WO/2019/078263A1 |
The present invention addresses the problem of providing a method for easily and efficiently preparing a functional and matured neuromuscular junction (NMJ) in vitro. This problem is solved by a method for producing an artificial neuromu...
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WO/2019/071353A1 |
A method of reducing glutamate efflux from skeletal muscle by inhibiting system Xc- activity is provided. The method is useful for the treatment of statin-induced myalgia. Pharmaceutical compositions useful to treat statin-induced myalgi...
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WO/2019/073507A1 |
Provided is a medicinal composition for preventing and/or treating amyotrophic lateral sclerosis (ALS), said composition containing a plexin A1 antagonist.
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WO/2019/068888A1 |
The present invention relates to the treatment of subjective tinnitus. More precisely, it concerns the administration of botulinum toxin into the scalp, neck and face muscles, especially in the temporalis muscle, of patients suffering fr...
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WO/2019/071147A1 |
The disclosure relates to methods and compositions including p38 kinase inhibitors and agents that regulate expression of DUX4 and downstream genes including but not restricted to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDClL, RFPL2, CC...
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WO/2019/068604A1 |
The present 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 for use in the treatment of spinal muscular atrophy (SMA), its pharmaceutical composition to be...
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WO/2019/071144A1 |
The present invention relates to compositions and methods of inhibiting p38 kinase to reduce gene and protein expression of DUX4 and downstream genes regulated by DUX4. The present invention further relates to methods for treating patien...
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WO/2019/067979A1 |
The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal anti- inflammatory compound.
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WO/2019/065928A1 |
The present invention addresses the problem of providing a compound for prophylaxis and/or treatment of inflammatory diseases of the central nervous system, or a pharmacologically acceptable salt thereof. Provided is a compound represent...
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WO/2019/067981A1 |
The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal antiinflammatory compound.
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WO/2019/065791A1 |
Provided is a compound which has an MAGL inhibitory effect and is expected to be useful as a prophylactic or therapeutic agent for neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, Hutchinson's disease, amyotrop...
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WO/2019/067975A1 |
The present disclosure relates to methods of treating Duchenne's Muscular Dystrophy by administering an antisense oligonucleotide that induces exon skipping and a non-steroidal anti- inflammatory compound.
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WO/2019/062662A1 |
Disclosed are a substituted pyrimidine piperazine compound of Formula (I), or a stereoisomer, a geometric isomer, a tautomer, an N-oxide, a solvate, a metabolite, a pharmaceutically acceptable salt or a prodrug thereof, and a pharmaceuti...
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WO/2019/060297A1 |
Described herein are methods for mitochondrial transplantation wherein old, damaged, or mutated mitochondria are replaced with young, undamaged, or non-mutated mitochondria. The old, damaged, or mutated mitochondria can also be replaced ...
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WO/2019/059973A1 |
Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
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WO/2019/058132A1 |
This invention relates to compounds that are inhibitors of the CDK12 kinase. The compounds are useful in the treatment of disorders mediated by CDK12 kinase including myotonic dystrophy type 1 (DM1) and other disorders caused by the gene...
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WO/2019/055832A1 |
Provided herein are compounds of Formula (I) and (II) and their salts, and compositions comprising such compounds that are useful for useful for modulating neutral sphingomyelinase 2 (n-SMase2) in cells. Also disclosed herein are methods...
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WO/2019/054106A1 |
A suppressor of extracellular matrix production by epidermal growth factor receptor (EGFR)-expressing cells, said suppressor comprising an EGFR inhibitor as an active ingredient.
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WO/2019/054430A1 |
The purpose of the present invention is to provide a compound having an anti-inflammatory action, a pharmacologically acceptable salt thereof, etc. A solving means of the present invention is a compound of general formula (1) or a pharma...
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