Even with novel technologies like gene therapy, gene editing, or mRNA, each must address a specific gene or disease ... methylmalonic acidaemia (MMA) and phenylketonuria (PKU). One of the interesting ...

PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria ... muscular dystrophy for the first time. PKU is a rare genetic disease that manifests at ...

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 ...