Katie - Living with PKU — Katie, Living with PKU, United States

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.

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Our Scientific Platforms

Splicing

Modulating splicing to control protein production

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Inflammation/ Ferroptosis

Targeting oxidative stress and inflammation pathways

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Rare Journeys

Discover stories from the rare disease community.

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Therapeutic Areas

Diseases in focus

Phenylketonuria (PKU)

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

Aromatic L-amino acid decarboxylase (AADC) deficiency

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

Friedreich’s ataxia (FA)

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

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Resources For

Healthcare Providers

PTC works closely with healthcare providers to support their needs

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