[EN] NOVEL COMPOUNDS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS AND/OR DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM<br/>[FR] NOUVEAUX COMPOSÉS POUR LA PRÉVENTION ET/OU LE TRAITEMENT DES TROUBLES DE STOCKAGE DES LYSOSOMES ET/OU DES TROUBLES DÉGÉNÉRATIFS DU SYSTÈME NERVEUX CENTRAL
申请人:AMICUS THERAPEUTICS INC
公开号:WO2013148103A1
公开(公告)日:2013-10-03
Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.
描述了化合物(3R,4R,5S)-5-(二氟甲基)哌啶-3,4-二醇的新型盐,以及使用该化合物预防和/或治疗溶酶体贮积病和/或中枢神经系统退行性疾病的方法。具体来说,本发明提供了预防和/或治疗高雪氏病和/或帕金森病的方法。