Vertex Announces FDA-Approval of New Triple Drug Compound Effective for the Most Common Mutation in Cystic Fibrosis Yesterday, the CF Foundation and Vertex announced that the FDA had approved a triple-drug compound that is designed to be effective for the most common—and most severe—mutation in cystic fibrosis (CF). The new
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Advocacy
PCD Foundation Call to Action on Graham-Cassidy
The proposed Graham-Cassidy bill is bad for individuals with rare disorders. Our colleagues at the United Mitochondrial Disease Foundation (UMDF) engage an independent legislative advisory group, Holland & Knight, to assess the impact of proposed legislation on the UMDF community. UMDF has generously shared Holland & Knight’s overview of Graham-Cassidy