A CFTR CORRECTOR FOR THE TREATMENT OF GENETIC DISORDERS AFFECTING STRIATED MUSCLE
申请人:UNIVERSITA' DEGLI STUDI DI PADOVA
公开号:US20150328217A1
公开(公告)日:2015-11-19
The present invention relates to the use of CFTR correctors in the treatment of genetic disorders affecting striated muscle selected from sarcoglycanopathies, Brody's disease (BD) and the recessive forms of Cathecolaminergic Polymorphic Ventricular Tachycardia (CPVT).