Evrysdi (Risdiplam)

About Roche

Roche is a global healthcare leader with a unique dual-pillar strategy combining its Pharmaceuticals and Diagnostics divisions to address the full patient journey. Under CEO Thomas Schinecker, the company focuses on preventing disease through early identification, halting progression via targeted therapies, and restoring health with curative medicines. With a market cap of ~$256B, it leverages its integrated platform to drive innovation in oncology, neurology, immunology, and infectious diseases, while pursuing sustainable, long-term growth.

About PTC Therapeutics

PTC Therapeutics is a patient-centric, commercial-stage biopharma focused on discovering, developing, and commercializing clinically differentiated medicines for rare diseases. The company has achieved multiple regulatory firsts, including the first global treatment for Duchenne muscular dystrophy (Translarna), the first brain-infused gene therapy (Upstaza), and the first small molecule splicing modifier for spinal muscular atrophy (Evrysdi). Its strategy leverages two proprietary scientific platforms—Splicing and Inflammation & Ferroptosis—to build a diversified pipeline, supported by a global commercial infrastructure aimed at sustainable growth and expanding patient access.

Therapeutic Areas

Other Spinal Muscular Atrophy Drugs