Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
申请人:Amicus Therapeutics, Inc.
公开号:US11033538B2
公开(公告)日:2021-06-15
The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.
本发明提供了向有需要的受试者施用药理伴侣的剂量方案。这些给药方案可用于治疗由蛋白质错误折叠引起的疾病,如溶酶体贮积症。