European Medicines Agency (EMA) | What's new Follow Orphan designation: phosphorodiamidate morpholino oligonucleotide against the CUG repeat expansion of the DMPK gene mRNA transcript, conjugated to a cell penetrating peptide Treatment of dystrophic myotonia, 18/07/2025 Positive https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3087 ema.europa.eu
