Posts by: David Melamed

News

A specific variation in a gene associated with phenylalanine transport is strongly linked to weight gain and an increased risk of obesity in infants with phenylketonuria (PKU), a recent study ... Read more

April 1, 2020April 1, 2020

FDA Grants Orphan Drug Designation to APR-OD031 for PKU Treatment

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APR-OD031, an investigational treatment for people with phenylketonuria (PKU). This designation is intended to promote the development of ... Read more

March 25, 2020March 25, 2020

Rubius Stops Clinical Development of RTX-134 Cell Therapy for Phenylketonuria

News

Rubius Therapeutics is discontinuing its Phase 1b trial investigating RTX-134, a potential cell therapy for people with phenylketonuria (PKU). The decision comes as the company shifts its focus to the ... Read more