The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation...
Ultragenyx Pharmaceutical Inc. announced that the European Commission (EC) has approved the Marketing Authorization Application (MAA) for Mepsevii (vestronidase alfa),...
UX003-CL202 is an open-label, multi-center extension study to assess long-term safety and efficacy of UX003 treatment in patients with MPS 7.