PTC eyes filings for PKU therapy after phase 3 win

PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli

Synlogic preps phenylketonuria drug for phase 3 after mid-st...

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.

FDA slaps clinical hold on BioMarin's PKU gene therapy

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 (PK

Girl loses High Court battle over NICE review of BioMarin’s ...

Child with rare disease takes NICE to court over access to l...

NICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family claimed the NHS cost-effectiveness body is unlawfully limiting access to a lif