Pam Kowalcyzk and her daughter have PKU, a rare disorder that prevents the body from processing protein ...

“Lifting the Limits for PKU” raised $350,000 at an event held Sept. 5 at the Secret Garden in Rancho Santa Fe. The event raises funds to support NPKUA (National PKU Alliance) whose mission “is to ...

When her newborn was diagnosed with Phenylketonuria (PKU), Jewelia Belcher tells PEOPLE it felt like her “world was crashing down” ...

(RTTNews) - Eton Pharmaceuticals, Inc. (ETON), Tuesday announced final readout from the study of PKU GOLIKE as a protein substitute for the treatment of patients with phenylketonuria (PKU) during ...

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 ...

BEDFORD, Mass., March 15, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced two presentations during the ACMG Annual Clinical Genetics Meeting, ...

—According to a new systematic review of the literature, despite early and continuous dietary management to lower phenylalanine levels in patients with phenylketonuria (PKU), cognition may still be ...

Pam Kowalczyk (left) with her daughter, Kelsey, in August 2025. Growing up with a rare genetic condition, Pam Kowalczyk felt like no one understood what she was going through. All of that changed when ...