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Matches 51 - 100 out of 23,740

Document Document Title
WO/2018/053653A1
The present matter relates to compositions comprising Teneurin C-terminal Associated Peptide – 1 (TCAP-1) and methods and uses of same for enhancing and/or restoring muscle function.  
WO/2018/053632A1
Methods for modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift or nonsense mutation within the dystrophin gene. The methods comprise introducing a first cut within...  
WO/2018/056871A1
The invention relates to the field of molecular medicine. The subject matter of the invention is a use of oligonucleotide analogues, where one or more phosphodiester groups are substituted with a phosphoryl guanidine group, and derivativ...  
WO/2018/052114A1
The present invention pertains to a pharmaceutical composition which contains a morphinan derivative exhibiting the action of an opioid δ receptor agonist. By administering the pharmaceutical composition provided by the present inventio...  
WO/2018/051355A1
Methods of treating Duchenne muscular dystrophy (DMD), as well as methods of reducing the dose of steroids used to treat DMD are provided. Kits comprising tamoxifen and citrulline, arginine or a combination thereof, are also provided.  
WO/2018/052082A1
This Lin28a activator includes, as an active ingredient, a polysaccharide which contains at least one substance selected from the group consisting of ribose, xylose, and derivatives of each. A monocyte dedifferentiation inducer includes,...  
WO/2018/053004A2
Pharmaceutical compositions that stabilize a Clostridial toxin active ingredient are described. The compositions can be liquid or solid compositions, and comprise a surfactant and an antioxidant. In some embodiments, the compositions com...  
WO/2018/053280A1
The subject invention provides a method for treating a subject afflicted with Rett syndrome comprising administering to the subject an effective amount of pridopidine so as to thereby treat the subject.  
WO/2018/053004A3
Pharmaceutical compositions that stabilize a Clostridial toxin active ingredient are described. The compositions can be liquid or solid compositions, and comprise a surfactant and an antioxidant. In some embodiments, the compositions com...  
WO/2018/047894A1
Provided is a novel antibody. According to the present invention, a monoclonal antibody or a binding fragment thereof binds to domain 3 of a human LAG-3, and has the properties described in (i) and (vi) and one or more of the properties ...  
WO/2018/041942A1
Embodiments of the invention are directed to new polymer linked multimeric guanosine-3', 5'-cyclic monophosphate (cGMP) analogues that modulate the cGMP-signaling system, preferably having activating properties, and more preferably being...  
WO/2018/041563A1
The present invention relates to novel substituted phenoxy- and benzyloxy-piperidine compounds of formula (I) having P2X7 receptor (P2X7) antagonistic properties, pharmaceutical compositions comprising these compounds, chemical processes...  
WO/2018/042141A1
The present invention concerns a tryptophan metabolite chosen from kynurenic acid, anthranilic acid, quinolinic acid, picolinic acid, quinaldic acid and the mixtures of same or an enantiomer, diastereoisomer, hydrate, solvate, tautomer, ...  
WO/2018/043694A1
The present invention provides a composition for improving muscular endurance, which is composed of nutrients, can be ingested safely and can improve muscular endurance easily within a short time and with high efficiency. The composition...  
WO/2018/040627A1
Use of catalpol in preparation of a drug for preventing and treating or delaying the onset of myasthenia and/or amyotrophy, including myasthenia and/or amyotrophy caused by diabetes, and myasthenia and/or amyotrophy caused by defect of d...  
WO/2018/043714A1
The purpose of the present invention is to efficiently produce microglia from a pluripotent stem cells. Provided is a method for producing microglia from pluripotent stem cells, comprising the following steps: (a) a step of co-culturing ...  
WO/2018/042330A1
Methods of improving physical performance and endurance include administering a capsicum composition to a subject. More particularly the methods include administration of an effective amount of a capsicum composition including capsaicino...  
WO/2018/043476A1
Provided is a therapeutic agent for amyotrophic lateral sclerosis, comprising: a compound represented by formula (1) (in formula (1), R1 each independently represents an alkyl group containing 1-6 carbon atoms or a 4-hydroxyphenethyl gro...  
WO/2018/038265A1
The present invention provides: a novel use of a specific bicyclic nitrogenated heterocyclic compound as a PDE7 inhibitor; a novel bicyclic nitrogenated heterocyclic compound having a PDE7 inhibition activity, a method for producing the ...  
WO/2018/038255A1
The purpose of the present invention is to provide crystals of a compound that exhibits an analgesic action against neuropathic pain and/or fibromyalgia, the crystals being useful as pharmaceuticals. The present invention provides crysta...  
WO/2018/038966A1
The present invention provides a pharmaceutical composition comprising a formulation of a corticosteroid and at least one organic acid and, optionally, a fast-acting insulin analog, in a vehicle. Also provided are methods to induce the p...  
WO/2018/034334A1
Provided is a therapeutic agent which effectively acts on diseases associated with abnormalities in dystroglycan sugar chain modification. A method for diagnosing diseases associated with abnormalities in dystroglycan sugar chain modific...  
WO/2018/030432A1
Provided is a peptide having a short chain length, e.g. 20 amino acid residues or fewer, and exhibiting high myostatin inhibitory activity. The present invention is a peptide comprising an amino acid sequence represented by formula (1) i...  
WO/2018/026776A3
Disclosed herein, inter alia, are FOXMl modulators and methods for modulating the activity of androgen receptors in neuronal cells to treat various diseases, such as spinal-bulbar muscular atrophy, amyotrophic lateral sclerosis, and Alzh...  
WO/2018/019244A1
The present invention relates to salts of 2,6-dimethylpyrimidone derivative and uses of the salts. The present invention also relates to a pharmaceutical composition comprising the salts or a combination thereof, and uses of the salts or...  
WO/2018/017726A1
Disclosed herein are methods and compositions for alleviating side effects of statin administration, such as myopathic or myalgic side effects, short-term memory loss, abnormal liver function, glucose intolerance, hyperglycemia, increase...  
WO/2018/012582A1
Provided is a blood amino acid level elevation promoter which contains a whey protein hydrolysate as an active ingredient. The elevation of a blood amino acid level in a subject can be promoted by feeding the promoter to the subject.  
WO/2018/010965A1
Embodiments of the invention are directed to new equatorially modified polymer linked multimers of guanosine-3', 5'-cyclic monophosphate (cGMP) analogues that inhibit the cGMP-signaling system. The invention is also directed to related m...  
WO/2018/013477A1
This document provides methods and materials for activating muscle remodeling. For example, methods and materials for using GAR1 inhibitors to activate muscle remodeling are provided.  
WO/2018/012004A1
[Problem] The purpose is to provide a "functional agent having a dynapenia-suppressing function" having as an active ingredient components isolated and obtained from leaves or flowers of a specific plant raw material by a specific method...  
WO/2018/007475A1
The current invention provides antisense splice-switching oligonucleotides with improved characteristics that enhance clinical applicability for treating, ameliorating, preventing, and/or delaying neuromuscular disorders, such as DMD.  
WO/2018/009544A1
The present disclosure provides compounds of Formula (I), (II), and (III). The provided compounds are able to bind protein kinases (e.g., SIK) and may be useful in modulating (e.g., inhibiting) the activity of a protein kinase (e.g., SIK...  
WO/2018/009609A1
The present disclosure relates to crystalline solid forms of a stimulator of soluble guanylate cyclase (sGC), Compound I. Also provided herein are methods for the preparation of these solid forms. The invention also relates to pharmaceut...  
WO/2018/008749A1
A nucleic acid which can inhibit expression of the MEX3B gene with low cytotoxicity as a side effect, an MEX3B gene expression inhibitor which contains said nucleic acid, a method for inhibiting MEX3B gene expression, and a prophylactic ...  
WO/2018/003962A1
2- (Piperidin-1-yl) pyrimidin-4 (3H) -ones characterized by having a 1,8-diazaspiro [4.5] deca-3-ene, 1-oxa-8-azaspiro [4.5] deca-3-ene, 2,8-diazaspiro [4.5] deca-3-ene, 2-oxa-8-azaspiro [4.5] deca-3-ene, 2,9-diazaspiro [5.5] undeca-3-en...  
WO/2018/000098A1
A method of treating an inflammatory myopathy includes topically administering to the skin of an individual with the inflammatory myopathy a composition comprising R-salbutamol, a physiologically acceptable derivative thereof, or a pharm...  
WO/2018/005805A1
Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 45 skipping are described.  
WO/2017/222040A1
The present invention provides a pharmaceutical composition which can be administered to a human body safely and is effective for the treatment of PolyQ diseases. The present invention provides: a pharmaceutical composition which contain...  
WO/2017/221883A1
[Problem] The present invention provides an antibody-drug conjugate (ADC). The present invention also provides a conjugate of an anti-CD71 antibody or an antigen-binding fragment thereof with a drug. The present invention further provide...  
WO/2017/218825A1
DNA-targeted nanocarriers for encapsulating an active agent and delivering it to extracellular DNA are provided. The nanocarriers, for example, polymeric particles, liposomes, and multilamellar vesicles have targeting moiety that targets...  
WO/2017/218592A1
The present disclosure relates to the treatment of muscle conditions, such as SMA, with the use of an agent that inhibits myostatin signaling. The disclosure includes combination therapies that include a myostatin inhibitor and a neurona...  
WO/2017/217525A1
The disclosure provides anti-myostatin antibodies and methods of making and using the same. Nucleic acids encoding the anti-myostatin antibodies and host cells comprising the nucleic acids are also provided. The disclosure also provides ...  
WO/2017/217556A1
The purpose of the present invention is to provide a medicine for preventing or treating TGFβ-related diseases by inhibiting signal transduction of TGFβ. Provided are: a TGFβ inhibitor that contains, as an active ingredient, the REIC/...  
WO/2017/217439A1
The purpose of the invention is to provide a compound that inhibits the binding of the ALS-related mutant SOD1 and Derlin-1, a drug containing the compound, and a method for treating ALS by administering the drug to a patient. More speci...  
WO/2017/211274A1
Provided are the use of lipids bearing fatty acids with an odd number of carbon atoms as pharmaceuticals or nutritional supplement. In particular, such lipids are used in the treatment and/or prevention of neurodegenerative diseases, opt...  
WO/2017/211303A1
Provided are certain pyrazine derivatives (I) as SHP2 inhibitors which is shown as formula (I), their synthesis and their use for treating a SHP2 mediated disorder. More particularly, provided are fused heterocyclic derivatives useful as...  
WO/2017/210989A1
Disclosed are a phloroglucinol-oxytocin injection and a preparation method therefor. Each 10,000 ml injection contains 100 ml of an oxytocin solution and 0.2 g of phloroglucinol, and a 1 ml injection contains 10 units of the oxytocin sol...  
WO/2017/213854A1
Provided herein are pharmaceutical compositions comprising Eteplirsen. Also provided herein are methods of treating a muscle disease in a subject in need thereof, comprising administering to the subject a pharmaceutical composition of th...  
WO/2017/207678A1
Wnt-inducible signalling protein 1 (WISP1) or a fragment or variant thereof for use in maintaining or increasing muscle function and/or mass in a subject, and/or substantially preventing or reducing muscle wasting in a subject.  
WO/2017/207600A1
The present invention relates to olesoxime for use in the treatment of spinal muscular atrophy (SMA), its pharmaceutical composition to be used in the treatment of SMA, its methods of treatment thereof.  

Matches 51 - 100 out of 23,740