phenylketonuria Archives -

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA

FDA slaps clinical hold on BioMarin’s PKU gene therapy

https://pharmaphorum.com/news/fda-slaps-clinical-hold-on-biomarins-pku-gene-therapy/

An 11-year-old girl with the rare disease phenylketonuria (PKU) has lost a legal battle in the High Court

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

https://pharmaphorum.com/news/girl-loses-high-court-battle-over-nice-review-of-biomarins-pku-drug/

NICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family

Child with rare disease takes NICE to court over access to life-changing therapy

https://pharmaphorum.com/news/child-with-rare-disease-takes-nice-to-court-over-access-to-life-changing-therapy/

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