Translating Science

Transforming Lives

Delivering transformative medicines to children and adults with serious diseases of high unmet need.

Innovation, fueled by science and a passion for patients

PTC is a global biopharmaceutical company that discovers, develops, and commercializes clinically differentiated medicines for children and adults living with serious diseases of high unmet need. Our ability to identify innovative new therapies and globally commercialize products is the foundation that drives investment in a diversified pipeline.

Explore our science

Focused research platforms at work for patients

Splicing

Modulating splicing to control protein production

Learn more

Inflammation & Ferroptosis

Targeting oxidative stress and inflammation pathways to treat CNS diseases

Learn more

Rare Journeys

Discover stories from the rare disease community.

Visit the Hub

Our Products

Groundbreaking Science

Our approved therapies in the U.S. and other countries include a series of firsts: the first approved treatment for Duchenne muscular dystrophy in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. We continue to invest in a diverse pipeline for conditions with significant unmet need.

See our products

Clinical Trials

We are committed to making progress in rare disease through clinical trial research.

Learn more about PTC's Clinical Trials

Therapeutic Areas

Diseases in focus

Aromatic L-amino acid decarboxylase (AADC) deficiency

Patients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement

Duchenne muscular dystrophy (DMD)

Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood

Phenylketonuria (PKU)

Metabolic condition caused by mutations in PAH, a key digestive enzyme, that causes cognitive disabilities and seizures

Friedreich’s ataxia (FA)

Rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart

See all therapeutic areas

Join Our Team

Working as OnePTC

As One PTC, we feel the urgent call to help those with significant unmet needs. We are motivated to provide treatments and new possibilities for our patients.

Doing things that no one’s ever done before is hard. That doesn’t deter us.

If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.

View PTC careers

The Latest on PTC

Making headlines

See all Newsroom Articles See all Press Releases

PRESS RELEASE

PTC Therapeutics to Participate at Upcoming Investor Conferences

Today PTC announced that its executives will speak at three upcoming conferences in September.

Read More
PRESS RELEASE

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results

PTC announced a corporate update and financial results for the second quarter ended June 30, 2025.

Read More
PRESS RELEASE

PTC Therapeutics Announces FDA Approval of Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

PTC Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU.

Read More