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Update for the EU Duchenne Community

Translating Science

Transforming Lives

Extending life’s moments for children and adults living with rare diseases

Innovation, fueled by science and a passion for patients

PTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create new therapies that address the underlying cause of the disease and deliver on our promise to create more moments for those who count on us.

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Focused research platforms at work for patients

Splicing
Modulating splicing to control protein production
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Inflammation & Ferroptosis
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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Our Products

Challenging what's possible

Harnessing groundbreaking science for patients has led to many firsts – the first approved treatment for Duchenne in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. Our innovative pipeline continues to expand as we discover and develop treatments using the newest technologies available.

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Clinical Trials

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

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Medical research areas

Diseases in focus

AADCd Patient
Aromatic L-amino acid decarboxylase (AADC) deficiency
Patients with AADC deficiency lack dopamine, an essential neurotransmitter that controls movement
DMD Patient
Duchenne muscular dystrophy (DMD)
Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood
PKU Patient
Phenylketonuria (PKU)
Metabolic condition caused by mutations in PAH, a key digestive enzyme, that causes cognitive disabilities and seizures
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We love what we do

PTC employees are working to change patients’ lives every single day.

We are motivated by our patients and the drive for them to have more moments with their families, made possible in part by our research and therapies.
If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.

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PTC Lab Employees
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