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Phenylketonuria News

Related terms: PKU

FDA Approves Sephience (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria

WARREN, N.J., July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved Sephience™ (sepiapterin) for the treatme...

FDA Approves Palynziq (pegvaliase-pqpz) for the Treatment of Adults with Phenylketonuria

May 24, 2018 - The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU). Patients with PKU are...

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