PTC Therapeutics, Inc.

PTC Therapeutics is a biotech company that develops medicines for rare genetic and neurological diseases. They make drugs like Evrysdi for spinal muscular atrophy, Translarna and Emflaza for muscular dystrophy, and Upstaza (sold as Kebilidi) for a rare brain disorder. Most of their medicines treat conditions that affect children and adults with uncommon illnesses that few other companies focus on. The company also has experimental drugs in development for diseases like Huntington's disease and Friedreich's ataxia.