发明名称 Methods for treatment of Fabry disease
摘要 Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.
申请公布号 US9480682(B2) 申请公布日期 2016.11.01
申请号 US201514641707 申请日期 2015.03.09
申请人 Amicus Therapeutics, Inc. 发明人 Lockhart David;Castelli Jeff
分类号 A61K31/45;A61K31/445;A61K45/06;C07D211/46;G01N33/50 主分类号 A61K31/45
代理机构 Servilla Whitney LLC 代理人 Servilla Whitney LLC
主权项 1. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 150 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.
地址 Cranbury NJ US