Use of epigenome-modifying compounds for the treatment of genetic muscular diseases linked to a protein-conformational disorder

摘要

A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.

主权项

1. A method for treating a genetic muscular disease that is linked to a conformational disorder of at least one protein causing the cellular degradation of the protein, wherein said disease is selected from the group consisting of sarcoglycanopathies, dysferlinopathies, anoctaminopathies, and dystrophies associated with a Fukutin-Related Protein (FKRP) disorder, comprising: administering to a patient in need thereof an effective amount of at least one compound selected from the group consisting of phenylbutyrate suberoylanilide hydroxamic acid (SAHA) and 5azacytidine.