One of the winners of the 2026 ASGCT Catalyst Award, Kiran Musunuru, MD, PhD, delivered a sobering lecture on the legacy of the Baby KJ story and the real-world discussions with the FDA to streamline ...

Among serious illnesses the genetic screening could detect is retinoblastoma, a rare and highly curable cancer that develops ...

End-to-end pipelines and regulatory advances aim to expand gene editing to broad patient populations Ajay Gannerkote, ...

The US FDA has granted BioMarin Pharmaceutical Inc. approval for use of pegvaliase-pqpz (PALYNZIQ) for the treatment of phenylketonuria (PKU) in adolescents down to age 12 years. Pegvaliase-pqpz is ...

The above button links to Coinbase. Yahoo Finance is not a broker-dealer or investment adviser and does not offer securities or cryptocurrencies for sale or facilitate trading. Coinbase pays us for ...

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; ...

BioMarin Pharmaceutical is continuing to slim down its pipeline, this time ending work on a preclinical phenylketonuria (PKU) drug once touted as a potential successor to its approved med Palynziq.

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 ...

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 ...

Joie Chavis’ third child has arrived. The dancer gave birth to baby No. 3, her first with NFL star Trevon Diggs, she revealed on her Instagram Story Monday. Chavis shared a video compilation of her ...