Orphan designation: 2'-O-(2-Methoxyethyl) modified antisense oligonucleotide against MECP2 pre-mRNA Treatment of MECP2 duplication syndrome, 21/11/2025 Positive | European Federation of Internal Medicine

Submitted by Anonymous (not verified) on 10 April 2026 - 10:31

Orphan designation: 2'-O-(2-Methoxyethyl) modified antisense oligonucleotide against MECP2 pre-mRNA Treatment of MECP2 duplication syndrome, 21/11/2025 Positive

Link:

https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3152

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