Sanofi's Genzyme unit has moved a step closer to securing EU approval for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after scoring
PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f
With more than 6,000 different diseases comprising the rare disease (RD) landscape, connecting patients and practitioners with the expertise needed to achieve th