At PTC, we are committed to a single purpose: Extending life’s moments for patients with rare diseases and their loved ones.

Our mission is to provide access to best-in-class treatments for rare disease patients who have little to no treatment options. Whether our therapies grant hours, days, months or years, every small moment is an enormous win – so our greatest accomplishments are “measured by moments.”

This mission underpins our focus on the discovery, development and global commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders.

As a science-driven, global biopharmaceutical company, we combine our strong clinical and scientific expertise with dedication to using groundbreaking science and new technologies to find innovative ways to treat these diseases, which can give patients and their families more moments.

Since 1998, PTC has been a biopharmaceutical company focused on innovative small molecule and gene therapies for rare genetic disorders. We operate offices and/or research labs in more than 20 countries and occupy a business footprint in more than 50 countries around the world. We also enjoy strong commercial performance and a robust capital position.

PTC achieved historic breakthroughs in treating the rare disease Duchenne muscular dystrophy in 2003 and has continued to grow and evolve ever since. Through strategic acquisitions and unparalleled scientific research, our team of “brilliant minds” is “measured by moments” brought to patients and caregivers who face rare disease challenges like Duchenne, aromatic L-amino acid decarboxylase (AADC) deficiency, Huntington’s disease, spinal muscular atrophy and many more.