Article
The myotubular and centronuclear myopathy patient registry: a multifunctional tool for translational research.
Neuromuscular disorders : NMD - 1 Feb 2024
Bullivant Joanne, Sen Anando, Page Jess, Graham Robert J, Jungbluth Heinz, Schara-Schmidt Ulrike, Lynch Orla, Bönnemann Carsten, Hollander Aart den, Lennox Anne, Moat Dionne, Saegert Claudia, Amburgey Kimberly, Buj-Bello Ana, Dowling James J, Marini-Bettolo Chiara
Abstract excerpt
The Myotubular and Centronuclear Myopathy Registry is an international research database containing key longitudinal data on a diverse and growing cohort of individuals affected by this group of rare and ultra-rare neuromuscular conditions. It can inform and support all areas of translational research including epidemiological and natural history studies, clinical trial feasibility planning, recruitment for...
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