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PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Shots:The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & BrazilApproval was based on APHENITY study results & long-term extension data showing durable effects and…

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