“We are encouraged to see these positive data that build on that legacy ... making Palynziq available for younger people living with PKU.” According to BioMarin, detailed study results will ...

Palynziq functions by substituting the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with phenylalanine ... "We are encouraged to see these positive data that build on that legacy ...

BioMarin BMRN has announced positive results from the phase III PEGASUS study evaluating Palynziq (pegvaliase) in adolescents aged 12-17 years with phenylketonuria (PKU). The study achieved its ...

has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for Sephience™ (sepiapterin) to treat phenylketonuria (PKU ...

We are encouraged to see these positive data that build on that legacy ... making PALYNZIQ available for younger people living with PKU." PEGASUS is a Phase 3 multi-center open-label randomized ...

Such positive data was achieved in this late-stage study targeting adolescent patients ages 12 - 17 with PKU specifically. The reason this drug has already been approved to treat adults with this ...

Join Matt Maley live this Wednesday at 6 PM ET to see how he’s trading it. PKU is a rare, inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine.

"This positive opinion is great news for the PKU patient community in Europe for whom there remains a significant unmet medical need," said Matthew B. Klein, M.D., Chief Executive Officer of PTC ...

has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for Sephience™ (sepiapterin) to treat phenylketonuria (PKU) in ...