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 Roberta
Membro del Collaboration Group
;
Bianchini Elisa
Membro del Collaboration Group
;
Volpe Pompeo
Membro del Collaboration Group
;
Mascarello Francesco
Membro 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).
File in questo prodotto:
Non ci sono file associati a questo prodotto.
Pubblicazioni consigliate

Caricamento pubblicazioni consigliate

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11577/3297652
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus ND
  • ???jsp.display-item.citation.isi??? ND
social impact