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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.
Bjorn Lindvall; Elizabeth Luebbe; Anna Lusakowska; Radim Mazanec; Giovanni Meola; Lianna Orlando; Masanori P Takahashi; Stojan Peric; Jack Puymirat; Rakocevic-Stojanovic, Vidosava; Miriam Rodrigues; ...
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.; ...
The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia
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.; ...