Compounds for use in the treatment of dystrophinopathies

A method for the treatment or prevention of a dystrophinopathy in a patient in need thereof comprising the administration to said patient of a compound selected from the group consisting of Difenpiramide, Veliflapon, Amopyroquine, Cloricromen, Alpelisib, Benorilate, Xipamide, Fabomotizole and Altizide, or a pharmaceutically acceptable salt thereof.

A method for the treatment or prevention of a dystrophinopathy in a patient in need thereof comprising the administration to said patient of a combination comprising two or more agents selected from the group consisting of Difenpiramide, Veliflapon, Amopyroquine, Cloricromen, Alpelisib, Benorilate, Xipamide, Fabomotizole, Altizide, or a pharmaceutically acceptable salt thereof;

• ataluren, deflazacort, givinostat, pizuglanstat, ifetroban, vamorolone, idebenone, prednisone, prednisolone, 20-hydroxyecdysone, metformin and L-citrulline combination, MA-0211, tamoxifen, dextro epicatechin, exaluren, ARM-210, 2,6-disubstituted-pyiridazin-3 (2H)-ones, N-Benzyl-p-toluenesulphonamide, MP-101, MT-002, rimeporide, tetracosactide hexaacetate, elamipretide, KER-050, SRP-5051, TXA-127, PB-1023, pamrevlumab, eteplirsen, golodirsen, vitolarsen, casimersen, ATL-1102, NS-089, renadirsen, WVEN-531, fordadistrogene movaparvovec, GNT-004, AAV1-follistatin viral vector, delandistrogene moxeparvovec, AT-702, AAVrh74-GALGT2, SGT-001, CAP-2003, CAP-1002;
• wherein at least one of said agents is selected from the group consisting of Veliflapon, Difenpiramide, Amopyroquine, Cloricromen, Alpelisib, Benorilate, Xipamide, Fabomotizole and Altizide, or a pharmaceutically acceptable salt thereof.