North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up.
Giorgia CorattiMarika PaneClaudia BrognaValeria RicottiSonia MessinaAdele D'AmicoClaudio BrunoGian Luca VitaAngela BerardinelliElena MazzoneFrancesca MagriFederica RicciTiziana MonginiRoberta BattiniLuca BelloElena PegoraroGiovanni BaranelloPrevitali Stefano CarloLuisa PolitanoGiacomo P ComiValeria A SansoneAlice DonatiJean Yves HogrelVolker StraubSilvana De LuciaErik H NiksLaurent ServaisImelda De GrootMary ChesshyreEnrico BertiniNathalie M GoemansFrancesco MuntoniEugenio Mercurinull nullPublished in: PloS one (2021)
Our results confirm different progression of disease in subgroups of patients with deletions amenable to skip different exons. This information is relevant as current long term clinical trials are using the NSAA in these subgroups of mutations.