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This week in the Pharmcast, we caught up with Dr. Tim Franson to discuss the leading edge of rare disease drug development. 

Dr. Franson has been actively helping Parent Project Muscular Dystrophy, a Duchenne Muscular Dystrophy Patient Advocacy group, which is doing pioneering work collaborating with FDA to inform benefit-risk and establish FDA guidance.

Duchenne has been a great test case, so we sat down with Dr. Franson to share his lessons learned and understand what’s happening on the frontiers of rare disease drug development.

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