The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Apr. 12, 2022
Filed:
Jan. 10, 2019
Inserm (Institut National DE LA Sante ET DE LA Recherche Medicale), Paris, FR;
Universite D'evry-val-d'essonne, Evry, FR;
Genethon, Evry, FR;
Universita 'degli Studi Di Padova, Padua, IT;
Isabelle Richard, Evry, FR;
Dorianna Sandona, Padua, IT;
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MÉDICALE), Paris, FR;
UNIVERSITE D'EVRY-VAL-D'ESSONNE, Evry, FR;
GENETHON, Evry, FR;
UNIVERSITA 'DEGLI STUDI DI PADOVA, Padua, IT;
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.