Orphan designation: Adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region Treatment of inherited retinal dystrophies, 20/06/2023 Positive

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https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2788

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Orphan designation: Adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region Treatment of inherited retinal dystrophies, 20/06/2023 Positive

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Orphan designation: Adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region Treatment of inherited retinal dystrophies, 20/06/2023 Positive

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