Genotype-related respiratory progression in Duchenne muscular dystrophy-A multicenter international study.
Federica TruccoDeborah RidoutJoana DomingosKate MareshMary ChesshyrePinki MunotAnna SarkozyStephanie RobbRosaline QuinlivanMollie RileyColin WallisElaine ChanFrancois AbelSilvana De LuciaJean-Yves HogrelErik H NiksImelda de GrootLaurent ServaisVolker StraubValeria RicottiAdnan ManzurFrancesco Muntoninull nullPublished in: Muscle & nerve (2021)
The progression of respiratory dysfunction follows different patterns for specific genotype categories. This information is valuable for prognosis and for the evaluation of exon-skipping therapies.