Dynamine 2 inhibitor for the treatment of centronuclear myopathies

摘要

A dynamine 2 inhibitor for use in the treatment of a centronuclear myopathy, wherein the dynamine 2 inhibitor is selected from the group consisting of an antibody directed against dynamine 2, a nucleic acid molecule that interacts directly with a nucleic acid encoding dynamine 2, and a small molecule that inhibits the activity, expression or function of dynamine 2, wherein said small molecule is selected from the group consisting of (3,4-dihydroxybenzylidene) hydrazide of 3-hydroxynaphthalene acid -2-carboxylic, 3-hydroxy-N '- [(2,4,5-trihydroxyphenyl) methylidene] naphthalene-2-carbohydrazide, tetradecyltrimethylammonium bromide, 4- chloro-2 - ((2- (3-nitrophenyl) -1,3-dioxo-2,3-dihydro-1H-isoindol-5-carbonyl) -amino) -benzoic acid, 2-cyano-N-octyl-3- [1- (3- dimethylaminopropyl) -1H-indole- 3-yl] acrylamide, 3- (2,4-dichloro-5-methoxyphenyl) -2-sulfanylquinazolin-4 (3H) -one, N, N'- (propane-1,3-diyl) bis (7.8 -dihydroxy-2-imino-2H-chromene-3-carboxamide ), N, N '- (ethane-1,2-diyl) bis (7,8-dihydroxy-2-imino-2H-chromene-3-carboxamide), octadecyltrimethylammonium bromide, dynamine inhibitor peptide with the sequence of amino acids: 3-hydroxy-N '- [(2,4,5-trihydroxyphenyl) methylidene] naphthalene-2-carbohydrazide and 4- (N, N-dimethyl-N-octadecyl-N-ethyl) -4- bromide aza-10-oxatricyclo- [5.2.1] decane-3,5-dione.