Why 95% of Rare Diseases Still Have No Treatment
About 90–95% of rare diseases still lack an FDA-approved treatment, underscoring the urgency for continued innovation on behalf of the estimated 30 million Americans — and families — affected by these conditions
During Rare Disease Week, this special episode of the Making Medicine podcast focuses on the patients, caregivers, and policy choices shaping the future of rare disease research. Host John Stanford is joined by Lisa Schlager (FORCE), Elaine Towle (Prader-Willi Syndrome Association), and Josh Trent (Save Rare Treatments Task Force) for a thoughtful discussion on the scientific…
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