Neuromuscular disorders : NMDMyotonic DystrophyHumansRegistriesNetherlandsDisease Progression
287th ENMC international workshop: Harmonization and federated analysis of myotonic dystrophy registries to model heterogeneous disease trajectories. Hoofddorp, the Netherlands, 28-30 March 2025.
Leandre A la Fontaine, Daniël van As, Guillaume Bassez, Nicholas E Johnson, Catharina G Faber, Peter A C 't Hoen, 287th ENMC workshop participants
Published: 202610.1016/j.nmd.2025.106257
Abstract
The 287th ENMC International Workshop convened experts from ten countries to address the harmonization and federated analysis of Myotonic Dystrophy Type 1 (DM1) registries. With over 10,500 patients enrolled globally, registries remain fragmented, li…
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