BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold ...

Alexa Beichler's boys were born with classic phenylketonuria (PKU), a metabolic condition that requires them to be on medical-grade formula for life Anya Leon is a Senior News Editor and the Parents ...

Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed to show efficacy, putting its ...

Alexa Beichler's two younger sons, Jax, 6 months, and Taylor, 20 months, were born with classic phenylketonuria (PKU), a metabolic condition that requires them to be on medical-grade formula for life ...

Every two to four weeks, Connor Delaney pricks his finger with a 3-inch lancet. He takes the sample of blood and smears just enough into each of the designated circles onto the screening pamphlet.

Dinner at Kristi and Matt Smith's home in Tucson, Arizona, is always a bit of a production. As their boys rabble-rouse, the parents are meticulously measuring every ounce of food that will go on their ...

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.

QUESTION: I have PKU (phenylketonuria). Is any PKU research being done at all? ANSWER: Phenylketonuria research goes all the way back to the 1930s, when two children with mental retardation were ...

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 ...

Debbie Colyer was diagnosed with phenylketonuria (PKU) in 1961. In Australia, so little was known about the disorder, which can lead to permanent brain damage, that staff at Camperdown Children's ...