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Drisapersen

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An exon skipping experimental Medicine[1] [2] PRO051/GSK2402968 is considered an orphan drug Duchenne muscular distrophy

Links

Duchenne muscular dystrophy: Still an incurable disease

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Eteplirsen a 30-nucleotide phosphorodiamidate morpholino oligomer

References

References:
  1. ↑ https://web.archive.org/web/20120729012249/http://www.prosensa.eu/technology-and-products/pipeline/pro051gsk2402968
  2. ↑ https://www.whocc.no/atc_ddd_index/?code=M09AX04
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