Novel enzyme therapy will treat adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment. The US Food and Drug Administration (FDA) has approved biotech ...

These data provide further evidence of the potential benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients. Also Read: PTC Therapeutics Inks Licensing Pact With ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics ...

oral treatment for PKU. PKU is a genetic disease that manifests at birth and results from an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods.

The data suggest that a significant majority of PKU patients could benefit from dietary liberalization while maintaining blood phenylalanine (Phe) control with sepiapterin treatment. The APHENITY ...

At the end of the one-week treatment period, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to ...

Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria ...

Considering taking supplements to treat phenylketonuria pku? Below is a list of common natural remedies used to treat or reduce the symptoms of phenylketonuria pku. Follow the links to read common ...

The overall funding strategy of the NPKUA is to support projects that will promote advances in the treatment and management of PKU, with the long term goal of facilitating the development of a cure.