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Comprehensive Four-Year Disease Progression Assessment of Myotonic Dystrophy Type 1

Leandre A. la Fontaine, Johanna E. Bruijnes, Fran H. P. Smulders, Carla Gorissen-Brouwers, Ilse E. A. Karnebeek,Hilde M. H. Braakman,Sylvia Klinkenberg,Karlien Mul,Peter-Bram A. C. 't Hoen,Sander M. J. van Kuijk,Baziel G. M. van Engelen,Ingemar S. J. Merkies,Catharina G. Faber

NEUROMUSCULAR DISORDERS（2024）

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关键词
Outcome measures,Natural history,Myotonic dystrophy type 1,DM1,Trial readiness
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