FDA Releases Guidance on Treatment for Duchenne Muscular Dystrophy

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The agency issues draft guidance on the development of drugs to treat Duchenne muscular dystrophy.


FDA released draft guidance on June 9, 2015 that will assist drug companies in the clinical development of drugs to treat X-linked Duchenne muscular dystrophy (DMD) as well as Becker muscular dystrophy, DMD-associated dilated cardiomyopathy, and symptomatic carrier states in females. The guidance does not address treatments for secondary complications of muscle degeneration in dystrophinopathies.


In developing the draft guidance, FDA took into consideration a proposed draft guidance written by the Parent Project Muscular Dystrophy (PPMD) advocacy group as well as public comments to the PPMD guidance. FDA states that the collaboration with PPMD is an example of how input from stakeholders, patients, and caregivers can be beneficial to drug development. “FDA values PPMD’s effort and input and appreciates the insights provided by the DMD community.” 

Source: FDA.gov