Combination treatment of sarcoglycanopathies
AssigneesInserm (Institut National De La Sante Et De La Recherche Medicale), Universite D'Evry Val D'Essonne, Genethon, Universita' Degli Studi Di Padova
InventorsIsabelle RICHARD, Dorianna SANDONA
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Abstract
Sarcoglycanopathies are autosomal recessive diseases caused by mutations in the one of the genes coding for any sarcoglycans (SG). The inventors previously showed that the application of small molecules developed to rescue ΔF508-CFTR trafficking, and known as CFTR correctors, improved the maturation of several α-sarcoglycan mutants that were consequently rescued at the plasma membrane (WO 014086687). Now, the inventors show that some specific CFTR correctors provide additive and even synergic effect when administered in combination.
Claims4
A method of treating a sarcoglycanopathy in a patient in need thereof comprising administering to the patient a therapeutically effective combination of N-(2-(5-chloro-2-methoxyphenylamino)-4′-methyl-4,5′-bithiazol-2′-yl)pivalamide (C17) and a further compound that is selected from the group consisting of N-[2-(5-Chloro-2-methoxy-phenylamino)-4′-methyl-[4,5′]bithiazolyl-2′-yl]-benzamide (C4), 4,5,7-trimethyl-N-phenylquinolin-2-amine (C5), N-(4-bromophenyl)-4-methylquinolin-2-amine (C6) and N-(2-(3-acetylphenylamino)-4′-methyl-4,5′-bithiazol-2′-yl)benzamide (C13).
Dependencies are inferred from the claim text.