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Orphan designation - Europe
- Substance: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate aminotransferase gene
- ATC code : -
- EU Number: EU/3/12/974
- Designation date : 21/03/2012
- Sponsor of orphan designation : UNIQURE BIOPHARMA B.V.
- Summary of Opinion
Designation
Treatment of primary hyperoxaluria type 1.
Diseases list
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