Submitted by Anonymous (not verified) on 10 April 2026 - 10:50
Orphan designation: gildeuretinol, gildeuretinol acetate Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, 22/08/2025 Positive
Source:
Orphan designation: gildeuretinol, gildeuretinol acetate Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, 22/08/2025 Positive