The European Medicines Agency has OKd nine new medicines for EU-wide approval, but recommended against renewing the conditional marketing authorization of two drugs. 

PTC Therapeutics’ CEO Matthew Klein says the uncertainties of gene therapy development and commercialization have led to huge opportunity costs. After shelving PTC’s preclinical gene therapy programs, he is leveraging the company's diverse platforms and expanding its commercial efforts.

UniQure’s Phase I/II interim readout in Huntington’s disease showed improvements on functional and clinical measures, but mixed results on a pair of biomarkers.

The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.