Protein substitutes (PS) are an essential component in the dietary management of phenylketonuria (PKU ... products were available in each country/treatment center. Between countries, the ...

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 ...

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria ... At the moment the main treatment strategy is to restrict phe in the ...

NGGT Inc., ("Next Generation Gene Therapeutics" or "NGGT"), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced ...

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 ...

(NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU).

This advancement aims to enhance compliance, particularly among pediatric patients, who often struggle with the high volumes associated with existing sapropterin treatments. If approved, RLF-OD032 ...

WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of ...