Orphan designation: botaretigene sparoparvovec Treatment of inherited retinal dystrophies due to defects in the RPGR gene, 23/07/2025 Positive | European Federation of Internal Medicine

Submitted by Anonymous (not verified) on 9 September 2025 - 14:59

Orphan designation: botaretigene sparoparvovec Treatment of inherited retinal dystrophies due to defects in the RPGR gene, 23/07/2025 Positive

Link:

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1715

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