Newborn screening for PKU has allowed for early management of the condition, which serves to improve the prognosis in most cases. Research into PKU in adults has focused mostly on cognitive function; ...

Researchers in Germany recently evaluated executive functions in 36 patients who were diagnosed with PKU during childhood. Significant negative correlations were observed between elevated childhood ...

WARREN, N.J., Dec. 22, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that Sephience™ (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare ...

WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric ...

CAMBRIDGE, Mass., Jan. 30, 2020 (GLOBE NEWSWIRE) -- Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an ...

PTC Therapeutics Inc, a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare ...

The safety profile and overall efficacy trends in adolescents were consistent with the known profile of the medicine in adults. The vast majority of adverse events (AEs) in the study were manageable, ...

If you or a loved one has phenylketonuria, you may be considering treatment options. One medicine you might hear about is Palynziq (pegvaliase-pqpz). Phenylketonuria, or PKU for short, is a rare ...

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 ...

TORONTO, Dec. 10, 2025 /CNW/ - PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU) ...