The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

LNAA products are only recommended by the manufacturers for patients >8 years old, excluding maternal PKU patients. Denmark does not allow full LNAA treatment and semi-free diet before 18 years old.

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 ...

Codexis has pursued this path with the development of CDX-6114, an orally administered enzyme for the potential treatment of the orphan metabolic disorder phenylketonuria (PKU). PKU is an inborn ...

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 ...

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 WALNUT CREEK, Calif., Nov. 11, 2024 ...

as they show great promise for NGGT002 to serve as a safe and effective treatment for PKU," said Yiting Liu, Ph.D., VP of Translational Research at NGGT. "With our initial 40-week span of positive ...

Q3 2024 Earnings Call Transcript November 7, 2024 PTC Therapeutics, Inc. beats earnings expectations. Reported EPS is $-1.39, ...

Corwin’s optimism is further bolstered by the compelling clinical data demonstrating sepiapterin’s efficacy, allowing PKU patients ... on the development of new treatments for multiple ...