The present invention is directed to compounds and pharmaceutically acceptable salts thereof which are correctors of the cellular processing of cystic fibrosis transmembrane conductance regulator protein (hereinafter CFTR), for use 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).
A CFTR Corrector For The Teatment Of Genetic Disorders Affecting Striated Muscle
Sandonà Dorianna
;Sacchetto RobertaMembro del Collaboration Group
;Bianchini ElisaMembro del Collaboration Group
;Volpe PompeoMembro del Collaboration Group
;Mascarello FrancescoMembro del Collaboration Group
2018
Abstract
The present invention is directed to compounds and pharmaceutically acceptable salts thereof which are correctors of the cellular processing of cystic fibrosis transmembrane conductance regulator protein (hereinafter CFTR), for use 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).
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