FDA slaps clinical hold on BioMarin’s PKU gene therapyBioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA Share XFDA slaps clinical hold on BioMarin’s PKU gene therapyhttps://pharmaphorum.com/news/fda-slaps-clinical-hold-on-biomarins-pku-gene-therapy/
Girl loses High Court battle over NICE review of BioMarin’s PKU drugAn 11-year-old girl with the rare disease phenylketonuria (PKU) has lost a legal battle in the High Court Share XGirl loses High Court battle over NICE review of BioMarin’s PKU drughttps://pharmaphorum.com/news/girl-loses-high-court-battle-over-nice-review-of-biomarins-pku-drug/
Child with rare disease takes NICE to court over access to life-changing therapyNICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family Share XChild with rare disease takes NICE to court over access to life-changing therapyhttps://pharmaphorum.com/news/child-with-rare-disease-takes-nice-to-court-over-access-to-life-changing-therapy/
