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Putting Patients First: Recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy and other rare, serious and life-threatening neurologic disorders

PPF imageYears of significant investments in research and development have created a robust pipeline of compounds under investigation for the treatment of Duchenne muscular dystrophy (Duchenne). Today, at least six compounds are now in Phase 2 or Phase 3 clinical trials and approaching consideration for review and approval. Some patients are already benefiting from access to these investigational treatments, but many others – unable to meet the inclusion criteria – are left waiting.

The passage of the U.S. Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012 reinforced the need for the FDA to enhance patients’ access to new medical treatments, particularly with regard to those for rare, serious and life-threatening disorders such as Duchenne. In combination with the existing authority of the FDA, the new provisions in FDASIA present a real opportunity to speed responsible access to new therapies.

To ensure we make the most of these new opportunities, Parent Project Muscular Dystrophy (PPMD) brought together an expert Advisory Committee, including leading voices in academia, industry, and patient advocacy, to issue recommendations about how to effectively evaluate new therapies for Duchene and other, rare, serious, and life-threatening disorders. Read the white paper recommendations. For a brief synopsis of the white paper recommendations, see the executive summary.


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