European Medicines Agency (EMA) | What's new Follow Orphan designation: adeno-associated viral vector serotype 9 containing the human CTNNB1 gene Treatment of autosomal dominant polycystic kidney disease, 18/07/2025 Positive https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3101 ema.europa.eu
