[EN] COMPOSITIONS AND METHODS FOR TREATING KABUKI SYNDROME AND RELATED DISORDERS<br/>[FR] COMPOSITIONS ET MÉTHODES PERMETTANT DE TRAITER LE SYNDROME DE KABUKI ET LES TROUBLES ASSOCIÉS
申请人:UNIV JOHNS HOPKINS
公开号:WO2015184279A1
公开(公告)日:2015-12-03
A method is provided for treating a Mendelian disorder of the epigenetic machinery (e.g., Kabuki syndrome) in a subject in need thereof. In particular, the method comprises administering to the subject a ketogenic composition in an amount sufficient to produce a physiologically acceptable ketosis in the subject. A method for selecting a subject for treatment of a Mendelian disorder of the epigenetic machinery (e.g., Kabuki syndrome) is also provided. Ketogenic compositions and kits useful for practicing the methods are also provided.