Upstaza (eladocagene exuparvovec)

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.

Therapeutic Areas