申请人:Commissariat à l'Energie Atomique et aux Energies
Alternatives
公开号:EP3030657B1
公开(公告)日:2018-07-18
Compounds Having Activity in Correcting Mutant-Cftr Processing and Uses Thereof
申请人:Verkman Alan
公开号:US20080318984A1
公开(公告)日:2008-12-25
The invention provides compositions, pharmaceutical preparations and methods for correcting cellular processing (e.g., folding, trafficking, or post-translational modification) of a mutant-cystic fibrosis transmembrane conductance regulator protein (e.g., ΔF508 CFTR) that are useful for the treatment of cystic fibrosis (CF). The compositions and pharmaceutical preparations of the invention may comprise one or more aminobenzothiazole-containing compounds, aminoarylthiazole-containing compounds, quinazolinylaminopyrimidinone-containing compounds, bisaminomethylbithiazole-containing compounds, or phenylaminoquino-line-containing compounds of the invention, or an analog or derivative thereof.
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).
CFTR CORRECTOR FOR THE TREATMENT OF GENETIC DISORDERS AFFECTING STRIATED MUSCLE
申请人:UNIVERSITA' DEGLI STUDI DI PADOVA
公开号:US20180221347A1
公开(公告)日:2018-08-09
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).