Ultragenyx seeks marketing authorization for Ace-ER tablets in Europe
Ace-ER is designed for the replacement of the deficient sialic acid substrate in patients with GNE Myopathy. A defect in the biosynthetic pathway for sialic acid, with onset
Ray Therapeutics has received priority medicines (PRIME) designation from the European Medicines Agency (EMA) for its RTx-015 gene therapy to treat retinitis pigmentosa (RP).