Rare disease patients cheered by positive results from PTC trial

18 May 2023

Positive results from the Phase III APHENITY study have lifted shares in PTC Therapeutics (Nasdaq: PTCT) by around 8%.

The company is testing an oral formulation of synthetic sepiapterin as a treatment for adults and children with phenylketonuria (PKU), a rare and inherited metabolic disease which affects the brain.

PKU is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe).

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