Evrysdi (risdiplam)
Spinal Muscular Atrophy (SMA)
ApprovedCommercial
Key Facts
About PTC Therapeutics
PTC Therapeutics is a patient-centric, publicly traded biopharma leader dedicated to translating groundbreaking science into life-changing treatments for rare diseases. Its key achievements include commercializing a series of 'firsts' in rare disease treatment and building a diversified pipeline targeting neurological and metabolic disorders. The company's strategic direction is fueled by its deep expertise in two core scientific platforms—splicing modifiers and inflammation/ferroptosis inhibitors—and a robust global commercial infrastructure to support its mission of delivering transformative medicines worldwide.
View full company profileTherapeutic Areas
Other Spinal Muscular Atrophy (SMA) Drugs
| Drug | Company | Phase |
|---|---|---|
| Apitegromab (SRK-015) | Scholar Rock Holding | Phase 3 |
| Taldefgrobep alfa | Biohaven | Phase 3 |