BEDFORD, Mass., July 27, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (FIXX), a genetic medicines company, announced today encouraging initial clinical data from the first dose cohort in the ...

Claire can only eat 15g of protein a day due to PKU, a rare condition that causes a 'toxic' build-up of phenylalanine in the body.

Credit: Getty Images. The investigational therapy is an oral formulation of synthetic sepiapterin. Sepiapterin significantly reduced blood phenylalanine levels in adult and pediatric patients with ...

To control their phenylalanine (Phe) levels, patients with PKU are advised to follow a restricted diet. However, little is known about the long-term consequences. A new study of 15 adults with PKU ...

A MUM whose two kids have a rare disease which means eating could leave them with life-long brain damage is campaigning for ...

PHILADELPHIA— Phenylketonuria (PKU) is a rare newborn genetic disease that impacts between 1 in 10,000 to 1 in 20,000 people, depending on the individuals’ genetic ancestry. PKU causes an amino ...

Phenylketonuria (PKU) is a rare genetic condition that affects how your body processes an amino acid called phenylalanine (Phe). People with PKU have low levels or missing activity of an enzyme called ...

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 ...