Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30-31 January 2017

Straub V, Mercuri E, group DMDoms. Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30-31 January 2017. Neuromuscular disorders : NMD 2018;28:690-701   Introduction With the rapid increase in the number of interventional clinical trials in Duchenne muscular dystrophy (DMD) over the past few years, the need for a frank […]