Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals' genetic ancestry. PKU causes an amino acid—called ...
What Is Sephience, and Why Does It Matter? Sephience (sepiapterin) is a new oral medication approved by the FDA to treat a rare inherited condition known as phenylketonuria (PKU) in adults and ...
Sephience (sepiapterin) is a prescription drug that’s used along with a special diet to help lower phenylalanine levels in adults and certain children with phenylketonuria (PKU). Sephience comes as an ...
WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has ...
A Prescription Drug User Fee Act target date of February 28, 2026 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics ...
The U.S. Food and Drug Administration has approved Sephience (sepiapterin), an oral therapy by PTC Therapeutics, for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder that ...
Long-term data showed patients treated with sepiapterin maintained mean blood Phe control while significantly increasing protein intake. The Food and Drug Administration (FDA) has accepted for filing ...
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