Submitted by Anonymous (not verified) on 1 February 2024 - 10:02
Orphan designation: Adeno-associated viral vector serotype 1 containing the 3' portion of human OTOF gene, Adeno-associated viral vector serotype 1 containing the 5' portion of human OTOF gene Treatment of otoferlin gene-mediated hearing loss, 20/03/2023 Positive
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