Genetic modifiers of respiratory function in Duchenne muscular dystrophy.
Luca BelloGrazia D'AngeloMatteo VillaAurora FustoSara VianelloBeatrice MerloDaniele SabbatiniAndrea BarpSandra GandossiniFrancesca MagriGiacomo Pietro ComiMarina PedemontePaola TacchettiValentina LanzillottaFederica TruccoAdele D'AmicoEnrico BertiniGuja AstreaLuisa PolitanoRiccardo MassonGiovanni BaranelloEmilio AlbamonteElisa De MattiaFabrizio RaoValeria A SansoneStefano Carlo PrevitaliSonia MessinaGian Luca VitaAngela BerardinelliTiziana MonginiAntonella PiniMarika PaneEugenio MercuriAndrea VianelloClaudio BrunoEric P HoffmanLauren MorgenrothHeather Gordish-DressmanCraig M McDonaldnull nullElena PegoraroPublished in: Annals of clinical and translational neurology (2020)
These findings support GC efficacy in delaying respiratory insufficiency, and will be useful for the design and interpretation of clinical trials focused on respiratory endpoints in DMD.