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Matches 51 - 100 out of 25,447

Document Document Title
WO/2019/160148A1
The purpose of the present invention is to provide: a cell aggregate containing dopamine-producing neural precursor cells suitable for transplantation; a mixture thereof; and a method for preparing the same. This cell aggregate contains ...  
WO/2019/158075A1
The present invention provides a benzodiazepine derivative hydrochloride represented by formula I or a crystal structure of an ethanolate thereof, wherein R is methyl or ethyl. The present invention further provides a method for preparin...  
WO/2019/156252A1
The present invention provides a lysosomal disease preventive/therapeutic agent comprising an HMG-CoA reductase inhibitor.  
WO/2019/156199A1
The present invention addresses the problem of providing a novel drug for the prevention of autoimmune diseases, the suppression of the progression of the symptoms of autoimmune diseases, the suppression of the recurrence of autoimmune d...  
WO/2019/154329A1
The present invention relates to the field of pharmaceutical chemistry. Specifically, the present invention relates to a series of BET (bromodomain and extra-terminal domain) inhibitors having a novel structure, particularly inhibitors t...  
WO/2019/156465A1
The present invention relates to a pharmaceutical composition comprising (S)-2-(((4'-trifluoromethylbiphen-4-yl)methyl)amino)propanam ide methanesulfonate or a pharmaceutically acceptable salt thereof as an effective ingredient for preve...  
WO/2019/150119A1
Inhibitors of ubiquitin specific protease 19 (USP19) of Formula (I) are provided, together with pharmaceutical compositions comprising said inhibitors, and methods of use thereof. The compounds can be used in in the treatment of muscular...  
WO/2019/152416A1
Provided herein are compounds of Formula (I) and their salts, and compositions comprising such compounds that are useful for increasing the amount of NAD+ in cells. Also disclosed are methods of using the disclosed compounds and composit...  
WO/2019/151286A1
Provided is a topical dermatological composition having an inhibitory effect on collagen and fibronectin production. This topical dermatological composition contains pentosan polysulfate and/or a salt thereof as an active ingredient. Thi...  
WO/2019/151539A1
[Problem] To provide a method for efficiently incorporating a drug that is to function in muscle tissue, but when administered to an organism is itself not adequately incorporated into muscle tissue, into muscle tissue, particularly into...  
WO/2019/152549A1
Some embodiments of the methods and compositions provided herein relate to treating a subject suffering from hypertension, a cardiac injury, or a metabolic disorder. Some embodiments include administering an exosome to a subject. Some em...  
WO/2019/152820A1
Disclosed herein are methods of treating facioscapulohumeral muscular dystrophy (FSHD). In an example, a method of treatment includes administering to a subject an agent that increases NuRD and/or CAF-1 complex repression of DUX4, thereb...  
WO/2019/151287A1
Provided is an external composition for skin which has an effect of increasing skin blood flow. The external composition for skin according to the present invention comprises pentosan polysulfuric acid and/or a salt thereof as an active ...  
WO/2019/146642A1
This γ-aminobutyric acid derivative-containing tablet is characterized by not containing a silicic acid-based additive.  
WO/2019/146790A1
The present invention discloses: a food composition for ameliorating or preventing lifestyle-related diseases that includes fulvic acid as an active ingredient; a food composition for improving liver function that includes fulvic acid as...  
WO/2019/137922A1
The present invention relates to modulators of complex 1 protein q (C1q) that preferably mimic and/or stabilize the interaction of inhibitor apolipoprotein-E (ApoE) with C1q, and their uses in the prevention or treatment of C1q-related d...  
WO/2019/138012A1
Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ∆F508-CFTR t...  
WO/2019/135259A1
The present disclosure relates to a series of tricyclic compounds, their tautomers, polymorphs, stereoisomers, prodrugs, solvates, pharmaceutically acceptable salts, pharmaceutical compositions containing them and methods of treating con...  
WO/2019/136216A1
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. Here, compositions and methods for the treatment ...  
WO/2019/135363A1
The purpose of the present invention is to provide an effective therapeutic drug for various pathoses mainly involving tenosynovial lesions. Provided is a therapeutic drug comprising, as an active ingredient, tranilast or a pharmaceutica...  
WO/2019/132026A1
The present invention addresses the problem of providing a cell population including safe adhesive stem cells that maintain normal karyotypes, a production method therefor, and a pharmaceutical composition including said cell population....  
WO/2019/128120A1
Provided is a fully human neutralizing antibody combating tetanus toxin. Specifically provided is a monoclonal antibody combating tetanus toxoid, having heavy chain CDR1-3 regions as shown in SEQ ID NOs. 1-3 and light chain CDR1-3 region...  
WO/2019/131829A1
A delivery method is provided which delivers a gene modifying tool which achieves a high gene modification efficiency in cells. This composition, for inducing genetic modification in a target locus in a cell, contains 1) the compound rep...  
WO/2019/131798A1
Provided is a cell growth inhibitor containing, as an active ingredient, a compound represented by general formula (1) below, or a pharmacologically acceptable salt thereof. [In general formula (1), J1 and J2 represent CH or N, but both ...  
WO/2019/126647A1
The present application provides methods of treating a disease, such as Pompe disease, in a subject, comprising detecting an erythropoiesis biomarker in a sample of the subject after administration of methotrexate and a therapeutic agent...  
WO/2019/124355A1
Provided is fermented milk capable of promoting muscle synthesis in a subject. Muscle synthesis in a subject can be promoted by having the subject ingest fermented milk having a milk protein concentration of at least 1.9 mass%.  
WO/2019/121856A1
Compositions and methods can use a combination of an autophagy inducer, such as spermidine, and high protein for induction of autophagy in an individual in need thereof. Preferably, a formulation containing a combination of an autophagy ...  
WO/2019/121855A1
Compositions and methods can use high protein for induction of autophagy in an individual in need thereof. Preferably, a formulation containing protein in an amount effective to induce autophagy, for example in muscle, is administered to...  
WO/2019/122166A1
The present invention relates to the treatment of autonomic disorders with a clostridial neurotoxin comprising a Hcc domain from a BoNT/B, BoNT/D, BoNT/D-C, BoNT/F or BoNT/G, wherein the dose of the clostridial neurotoxin to be administe...  
WO/2019/115780A1
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...  
WO/2019/116256A1
A compound of formula (I) wherein R1, R2, R3, R4 χ γ anc| A are as defined herein. The compounds of the present invention are inhibitors of hematopoietic prostaglandin D synthase (H-PGDS) and can be useful in the treatment of Duchenne ...  
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...  
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...  
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.  
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...  
WO/2018/151841A8
Provided herein multispecific (e.g., bispecific) binding molecules comprising a first binding domain that binds an extracellular portion of dystroglycan and a second binding domain that binds laminin-2. Further provided herein are method...  
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...  
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...  
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...  
WO/2019/103109A1
The present invention provides a prophylactic and/or therapeutic agent for ALS that comprises an Src/c-Abl pathway inhibitor.  
WO/2019/067981A8
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.  
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...  
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...  
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.  
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...  
WO/2019/099800A1
Trophic factor expressing monocyte cells derived from gene-edited induced pluripotent stem cells, methods for making and using.  
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...  
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,...  
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...  
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, ...  

Matches 51 - 100 out of 25,447