Committee for Orphan Medicinal Products (COMP): 16-18 July 2024, European Medicines Agency, Amsterdam, the Netherlands, from 16 July 2024 to 18 July 2024

Submitted by Anonymous (not verified) on 3 October 2024 - 10:32

Committee for Orphan Medicinal Products (COMP): 16-18 July 2024, European Medicines Agency, Amsterdam, the Netherlands, from 16 July 2024 to 18 July 2024

Orphan designation: Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA Treatment of Leber's congenital amaurosis, 28/04/2016 Positive

Submitted by Anonymous (not verified) on 2 October 2024 - 15:40

Orphan designation: Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA Treatment of Leber's congenital amaurosis, 28/04/2016 Positive

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