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Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease.
Jorge Alberto Diaz Manera; Celine Dogan; Rasha El Sherif; Barbara Fossati; Caroline Graham; James Hilbert; Kristinia Kastreva; En Kimura; Lawrence Korngut; Anna Kostera-Pruszczyk; Christopher Lindberg; ...
The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
Joncourt,F.; Diaz-Manera,J.; Gallardo,E.; A.A. Karaduman; Topaloglu,H.; R. El Sherif; Stringer,A.; Shatillo,A.V.; A.S. Martin; H.L. Peay; Bellgard,M.I.; Kirschner,J.; Flanigan,K.M.; Straub,V.; Bushby,K.; ...