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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
den Bergen,J.C.; Rodrigues,M.; Roxburgh,R.; Lusakowska,A.; Kostera-Pruszczyk,A.; Zimowski,J.; Santos,R.; Neagu,E.; Artemieva,S.; Rasic,V.M.; Vojinovic,D.; Posada,M.; Bloetzer,C.; Jeannet,P.Y.; ...