Otsuka Pharmaceutical Development & Commercialization, Inc. and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced new ...

Growing up with a rare genetic disorder, Pam Kowalczyk often felt like no one truly understood her daily struggles. Years later, that sense of isolation changed in a life-altering way, when she ...

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 ...

Thanks to neonatal testing for phenylketonuria (PKU), children with PKU can develop normally through dietary modification. But less is known about health risks for adults with PKU. A new study set out ...

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