Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings.

ABSTRACT: In June 2010, 25 representatives from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the context of clinical trial design and analysis. The workshop was organized in response to a September 2009 European Medicines Agency meeting where a clear directive was given that an international consensus needs to be developed that provides a foundation for age-appropriate clinical outcome measures for use in clinical trials of emerging therapeutics for DMD. Data were presented from eight multicenter longitudinal datasets, representing nearly 1900 patients over a 20-year time period. This experience confirmed the feasibility of repeated evaluations performed at multiple sites and addressed several core issues in drug developme

SUBMITTER: Bushby K 

PROVIDER: S-EPMC3357954 | biostudies-literature | 2011 Sep

REPOSITORIES: biostudies-literature