Orphan designation: adeno-associated virus vector serotype SNY001 containing the human PAH gene Treatment of hyperphenylalaninaemia, 26/02/2025 Positive | European Federation of Internal Medicine

Submitted by Anonymous (not verified) on 11 March 2025 - 10:47

Orphan designation: adeno-associated virus vector serotype SNY001 containing the human PAH gene Treatment of hyperphenylalaninaemia, 26/02/2025 Positive

Link:

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3026

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