A Plea to the FDA to Give an Ultra-Rare Disease Drug a Fair Hearing

RARECast - A podcast by RARECast - Thursdays

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Shelley Bowen, co-founder and director of family services for the Barth Syndrome Foundation, about Barth syndrome, the fight over approval for the first drug to treat the condition, and why it points to a systemic problem with the lack of consistent use of the flexibility Congress granted the FDA to get treatments to patients with ultra-rare diseases.