[EN] COMPOSITIONS AND METHODS FOR THE TREATMENT OF FATTY ACID OXIDATION DISORDERS<br/>[FR] COMPOSITIONS ET MÉTHODES DE TRAITEMENT DE TROUBLES DE L'OXYDATION DES ACIDES GRAS
申请人:KANDULA MAHESH
公开号:WO2015028956A1
公开(公告)日:2015-03-05
The invention relates to the compounds of formula I or its pharmaceutical acceptable salts, as well as polymorphs, solvates, enantiomers, stereoisomers and hydrates thereof. The pharmaceutical compositions comprising an effective amount of compounds of formula I, and methods for the treatment of fatty acid oxidation disorders may be formulated for oral, buccal, rectal, topical, transdermal, transmucosal, intravenous, parenteral administration, syrup, or injection. Such compositions may be used to treatment of 2, 4 dienoyl coa reductase deficiency, carnitine/acylcarnitine translocase deficiency (cat), carnitine palmitoyl tranferase deficiency type i (cpt-i), carnitine palmitoyl tranferase deficiency type ii (cpt-2).
该发明涉及公式I的化合物或其药用可接受的盐,以及其多晶形、溶剂合物、对映体、立体异构体和水合物。包含公式I化合物的有效量的药物组合物,以及用于治疗脂肪酸氧化障碍的方法可以制备为口服、颊内、直肠、局部、经皮、经粘膜、静脉、肠内、糖浆或注射的给药途径。这些组合物可用于治疗2,4-二烯酰辅酶A还原酶缺乏症、肉碱/酰肉碱转运酶缺乏症(CAT)、肉碱棕榈酰转移酶缺乏症I型(CPT-I)、肉碱棕榈酰转移酶缺乏症II型(CPT-II)。