Last updated on 23-8-2019 by Anonymous (not verified)
Public Access
Published
Authors
Bladen,C.L.; Rafferty,K.; Straub,V.; Monges,S.; Moresco,A.; Dawkins,H.; A. Roy; Chamova,T.; Guergueltcheva,V.; Korngut,L.; Campbell,C.; Dai,Y.; Barisic,N.; Kos,T.; Brabec,P.; Rahbek,J.; Lahdetie,J.; Tuffery-Giraud,S.; Claustres,M.; Leturcq,F.; R. Ben Yaou; Walter,M.C.; O. Schreiber-Katz; Karcagi,V.; Herczegfalvi,A.; Viswanathan,V.; Bayat,F.; I. De La Caridad Sarmiento; Ambrosini,A.; Ceradini,F.; Kimura,E.; van den Bergen,J.C.; Rodrigues,M.; Roxburgh,R.; Lusakowska,A.; Oliveira,J.; Santos,R.; Neagu,E.; Butoianu,N.; Artemieva,S.; Rasic,V.M.; Posada,M.; Palau,F.; Lindvall,B.; Bloetzer,C.; A.A. Karaduman; Topaloglu,H.; Inal,S.; Oflazer,P.; Stringer,A.; Shatillo,A.V.; A.S. Martin; H.L. Peay; Flanigan,K.M.; Salgado,D.; von Rekowski,B.; Lynn,S.; Heslop,E.; Gainotti,S.; Taruscio,D.; Kirschner,J.; Verschuuren,J.; Bushby,K.; Beroud,C.; Lochmuller,H.Keywords
Abstract:
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the dystrophin protein. Although many novel therapies are under development for DMD, there is currently no cure and affected individuals are often confined to a wheelchair by their teens and die in their twenties/thirties. DMD is a rare disease (prevalence <5/10,000). Even the largest countries do not have enough affected patients to rigorously assess novel therapies, unravel genetic complexities, and determine patient outcomes. TREAT-NMD is a worldwide network for neuromuscular diseases that provi…