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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 ...

Credit: Getty Images. The investigational therapy is an oral formulation of synthetic sepiapterin. Sepiapterin significantly reduced blood phenylalanine levels in adult and pediatric patients with ...

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 ...

(RTTNews) - PTC Therapeutics, Inc. (PTCT), Thursday announced new data of the Phase 3 APHENITY trial and subsequent open-label extension study, evaluating the potential meaningful benefits of ...

The FDA approved sepiapterin (Sephience) for the treatment of children and adults with phenylketonuria (PKU), PTC Therapeutics announced. The approval includes broad labeling for the treatment of ...

What if we could help protect the kidneys of people with lupus without stopping their immune system? A group from the Medical University of South Carolina is looking into this possibility. They are ...

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Wednesday that the primary endpoint was achieved in the APHENITY, Phase 3 registration-directed clinical trial of sepiapterin in adult and pediatric ...

- First marketing authorization submission for sepiapterin with additional global submissions to follow in 2024 - SOUTH PLAINFIELD, N.J., March 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: ...

"The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide," said Matthew B. Klein, M.D., Chief ...

WARREN, N.J., March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at ...