SOM-1311 (SOM-0011) is under development for the treatment of phenylketonuria. The drug candidate is a repurposed drug and is pharmacological chaperone of phenylalanine hydroxylase. The drug candidate ...

The conditions selected for testing range from those for which the benefits of screening and treatment are beyond doubt—such as phenylketonuria and congenital hypothyroidism—to those for which ...

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 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 ...

today announced the full readout and compelling results from the clinical trial evaluating PKU GOLIKE as a protein substitute for the treatment of phenylketonuria (PKU) in patients during ...