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The Duchenne Platform Trial Stakeholder Meeting
September 9, 2019
The Duchenne Platform Trial is being designed to increase access to therapeutic trials across the Duchenne community, with the goals of:
- More rapid completion of testing and approval of new DMD therapies
- Reduced clinical trial start-up and execution time
- Enhanced patient experience, reduced number on placebo
- Potential for rapidly testing combination therapies
- The trial will study multiple drugs in multiple patient types with one common placebo-controlled arm.
The stakeholder meeting will be hosted by I-ACT for Children, Parent Project Muscular Dystrophy and the Critical Path Institute.
More details to come shortly!