PTC is committed to bringing hope to patients with rare and genetic diseases.
Our scientists are finding new ways to regulate biology to control disease.
We are developing new treatments for Duchenne Muscular Dystrophy, Cystic Fibrosis and other diseases.
Translating Science. Transforming Lives.
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PTC STRIVE Awards Program
Spinal Muscular Atrophy Program Advances into Phase 2 Clinical Studies in SMA Patients with RG7916
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