Last updated on 22-8-2019 by Anonymous (not verified)
Authors
Wood, Libby; Bassez, Guillaume; Corinne Bleyenheuft; Campbell, Craig; Cossette, Louise; Jimenez-Moreno, Aura Cecilia; Dai, Yi; Dawkins, Hugh; Manera, Jorge Alberto Diaz; Dogan, Celine; El Sherif, Rasha; Fossati, Barbara; Graham, Caroline; Hilbert, James; Kastreva, Kristinia; Kimura, En; Korngut, Lawrence; Anna Kostera-Pruszczyk; Lindberg, Christopher; Lindvall, Bjorn; Luebbe, Elizabeth; Anna Lusakowska; Mazanec, Radim; Meola, Giovani; Orlando, Liannna; Masanori P Takahashi; Peric, Stojan; Jack Puymirat; Rakocevic-Stojanovic, Vidosava; Rodrigues, Miriam; Roxburgh, Richard; Schoser, Benedikt; Segovia, Sonia; Shatillo, Andriy; Thiele, Simone; Tournev, Ivailo; van Engelen, Baziel; Vohanka, Stanislav; Lochmüller, HannsKeywords
Article written during project(s) :
Abstract:
BACKGROUND: Myotonic Dystrophy is the most common form of muscular dystrophy in adults, affecting an estimated 10 per 100,000 people. It is a multisystemic disorder affecting multiple generations with increasing severity. There are currently no licenced therapies to reverse, slow down or cure its symptoms. In 2009 TREAT-NMD (a global alliance with the mission of improving trial readiness for neuromuscular diseases) and the Marigold Foundation held a workshop of key opinion leaders to agree a minimal dataset for patient registries in myotonic dystrophy. Eight years after this workshop, we su…