PTC Therapeutics ^Public

PTC Therapeutics is a biopharmaceutical company that specializes in the discovery, development, and commercialization of small-molecule drugs. They focus on treating rare diseases such as Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Huntington's Disease, and Leukemia. With a track record of groundbreaking achievements, PTC Therapeutics has developed the first approved treatment for Duchenne Muscular Dystrophy, the first approved gene therapy infused into the brain, and the first approved small molecule splicing modifier. Their commitment to innovation and utilizing the newest technologies has allowed them to expand their pipeline and continue making progress in the field of rare disease treatments.