PTC Therapeutics | Pioneers in Rare Disease Treatments
PTC Therapeutics pioneers pharmaceutical science to transform lives and treat rare diseases, like Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Huntington's Disease, Leukemia, and more.
About PTC Therapeutics - Treatments for Rare Diseases
Learn about PTC Therapeutics, a global biopharmaceutical company. We are fueled by science, and committed to transforming the lives of patients with rare diseases.
Our Pipeline - PTC Therapeutics
PTC has been a pioneer in the development of breakthrough rare disease therapies, delivering a number of firsts for patients who previously had no other treatment options. Our strategy leverages our innovative scientific knowledge, our deep therapeutic expertise and robust global commercial infrastructure.
Our Science - PTC Therapeutics
We have focused our research efforts on two scientific platforms—splicing and inflammation and ferroptosis — where PTC has unique expertise to discover and advance to the clinic innovative therapies.
Newsroom | The Latest on PTC Therapeutics
Read the latest updates on innovations, research, rare disease treatments, business & finance, and community relations at PTC Therapeutics.
Investors | PTC Therapeutics, Inc.
Dec 19, 2024 · The Investor Relations website contains information about PTC Therapeutics, Inc.'s business for stockholders, potential investors, and financial analysts.
Press Releases - PTC Therapeutics, Inc.
WARREN, N.J. , Nov. 27, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million upon the closing of the transaction.
PTC Therapeutics Announces Vatiquinone NDA Submission to FDA …
WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to …
Therapeutic Areas - PTC Therapeutics
We are deeply committed to patients living with rare diseases who have little-to-no treatment options, and this passion fuels and inspires us. Our commercial and development efforts are focused in two therapeutic areas – neurology and metabolic conditions – in which we have deep experience and a record of success.
PTC Therapeutics Announces FDA Approval of AADC Deficiency …
WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain.