ARTICLE | Regulation
Patients lead the way
How a patient advocacy group led FDA to publish a draft guidance for DMD
June 15, 2015 7:00 AM UTC
FDA's draft guidance on Duchenne muscular dystrophy and related dystrophinopathies is the culmination of an extensive effort led by a patient advocacy group that enlisted the support of patients, caregivers, academia and industry.
DMD drug development has been hindered by a lack of defined outcome measures and understanding of surrogate markers or biomarkers that could support approval. To address this, FDA invited Parent Project Muscular Dystrophy (PPMD) and the DMD community to submit a proposed draft guidance that could clarify a clinical development pathway for approval. ...