Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease

Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease

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 cur...

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Bibliographic Details
Main Authors: Libby Wood, Guillaume Bassez, Corinne Bleyenheuft, Craig Campbell, Louise Cossette, Aura Cecilia Jimenez-Moreno, Yi Dai, Hugh Dawkins, Jorge Alberto Diaz Manera, Celine Dogan, Rasha el Sherif, Barbara Fossati, Caroline Graham, James Hilbert, Kristinia Kastreva, En Kimura, Lawrence Korngut, Anna Kostera-Pruszczyk, Christopher Lindberg, Bjorn Lindvall, Elizabeth Luebbe, Anna Lusakowska, Radim Mazanec, Giovani Meola, Liannna Orlando, Masanori P. Takahashi, Stojan Peric, Jack Puymirat, Vidosava Rakocevic-Stojanovic, Miriam Rodrigues, Richard Roxburgh, Benedikt Schoser, Sonia Segovia, Andriy Shatillo, Simone Thiele, Ivailo Tournev, Baziel van Engelen, Stanislav Vohanka, Hanns Lochmüller
Format: Article
Language:English
Published: BMC 2018-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Myotonic dystrophy
Registries
Clinical trials
Trial readiness
Online Access:http://link.springer.com/article/10.1186/s13023-018-0889-0

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http://link.springer.com/article/10.1186/s13023-018-0889-0

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