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Zoekresultaten - 3 results
Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease.
Richard Roxburgh; Benedikt Schoser; Sonia Segovia; Andriy Shatillo; Simone Thiele; Ivailo Tournev; Baziel van Engelen; Stanislav Vohanka; Hanns Lochmüller Source: Orphanet J Rare Dis, Volume 13, Issue ...
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.; ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
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.; ...