Protein substitutes (PS) are an essential component in the dietary management of phenylketonuria (PKU). PS are available as phenylalanine-free amino-acid mixtures (AAM), glycomacropeptide-based PS ...

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 ...

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 ...

Argentina has documented over 1,200 cases of inborn metabolic disorders annually, with phenylketonuria being the most ...

today reported positive topline results from its proof-of-concept clinical study evaluating RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, for the ...

The trial will continue to evaluate kidney ... acquisition of Boston-based Jnana Therapeutics and its clinical-stage oral phenylketonuria drug.

Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed to show efficacy ...

Relief plans to advance RLF-OD032 into a pivotal bioequivalence trial as part of the 505(b)(2) NDA submission process in the United States and pursuant to the development guidance previously ...

Beatty acknowledges the strength of the Phase 2 clinical trial data, which demonstrated ... for Sepiapterin for Phenylketonuria (PKU). Beatty’s analysis suggests a significant increase in ...

"The clinical trial data, including the evidence of enabling diet liberalization ... levels and has the potential to treat the broad range of PKU patients. About Phenylketonuria Phenylketonuria (PKU) ...