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

Straub_2018_Neuromuscul Disord. Aug;28(8);690-701

In January 2017 a workshop has been held, during which expert in the field discussed the currently available outcome measures, used in natural history studies and clinical trials. The validity and clinically relevance of outcome measures where discussed.

 

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 and honest discussion between those designing and using outcome measures has never been greater. In January 2017 clinicians, physiotherapists, imaging experts and patient advocacy group representatives came together for a two day workshop at The Foundry in London, UK, for a critical review of the different clinical outcome measures used to date in natural history studies and interventional trials for DMD. Participants were joined on the second day by regulatory and industry stakeholders. Sessions were prepared in advance of the workshop by working groups and were led by experts for specific outcome measures relevant to different stages of boys and men with DMD. The workshop focused on clinical and imaging outcome measures that are used as primary and secondary endpoints in trials and not on exploratory endpoints such as serum biomarkers.

 

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