a clinical-stage gene therapy candidate for the treatment of Phenylketonuria (PKU). "We are pleased with the results from our investigator-initiated trial, as they show great promise for NGGT002 ...

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 ...

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.

for the treatment of phenylketonuria (PKU). The four-way crossover pilot study compared the pharmacokinetics of Relief's investigational drug RLF-OD032 with the reference listed drug KUVAN ...

Titled "The Benefits of a Prolonged-Release Protein Substitute with Similarities to Natural Proteins as a Treatment for Phenylketonuria," the summary was co-authored by PKU specialists with insights ...

The NDA was submitted with an indication for the treatment of pediatric and adult patients with phenylketonuria (PKU), including all age groups and the full spectrum of disease subtypes.

The patents, based on these new findings, cover additional claims for Relief’s investigational drug RLF-OD032, a highly concentrated, novel liquid formulation of sapropterin dihydrochloride, for the ...

Pegvaliase-pqpz injection for subcutaneous use was approved by FDA in May 2018 for treating phenylketonuria (PKU), a rare genetic disease in adults. Pegvaliase-pqpz is a phenylalanine-metabolizing ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the <a ta ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria ... treatment for PKU," said Yiting Liu ...