PTC Therapeutics

Therapeutic Areas Diseases in focus Go to previous slide Go to next slide 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 Duchenne muscular dystrophy (DMD) Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood Frie

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Therapeutic Areas Diseases in focus Go to previous slide Go to next slide 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 Duchenne muscular dystrophy (DMD) Rare and fatal genetic disorder resulting in progressive muscle weakness from early childhood Frie

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