EMA Recommends Aqneursa for Niemann-Pick Type C Disease
Aqneursa: A Glimmer of Hope for Niemann-Pick Type C Disease? Examining the Future of Rare Disease Treatments The recent recommendation by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) to grant marketing authorization for Aqneursa (levacetylleucine) in the European Union marks a significant moment for individuals affected by Niemann-Pick type C … Read more