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Neurogene announces EMA grants orphan drug designation for aspartylglucosaminuria gene therapy
December 09, 2020Neurogene announces that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to its adeno-associated virus vector (AAV) with engineered transgene encoding the human AGA gene for patients diagnosed with aspartylglucosaminuria.
Pharma Sources Insight January 2025
