Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/01/2024 Positive | European Federation of Internal Medicine

Submitted by Anonymous (not verified) on 11 April 2024 - 12:58

Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/01/2024 Positive

Link:

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2888

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