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. 2020 Apr;30(2):67-70.
doi: 10.1089/nat.2020.0845. Epub 2020 Feb 11.

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

Annemieke Aartsma-Rus  1 , David R Corey  2   3
Affiliations

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

Annemieke Aartsma-Rus et al. Nucleic Acid Ther. 2020 Apr.
No abstract available

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Conflict of interest statement

A.A.R. discloses being employed by LUMC that has patents on exon-skipping technology, some of which have been licensed to BioMarin and subsequently sublicensed to Sarepta Therapeutics. As coinventor of some of these patents, A.A.R. is entitled to a share of royalties. A.A.R. further discloses being ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, CRISPR Therapeutics, Summit PLC, Alpha Anomeric, BioMarin Pharmaceuticals, Inc., Eisai, Astra Zeneca, Santhera, Audentes, Global Guidepoint and GLG consultancy, Grunenthal, Wave, and BioClinica, having been a member of the Duchenne Network Steering Committee (BioMarin) and being a member of the scientific advisory boards of ProQR, hybridize therapeutics, silence therapeutics, Sarepta Therapeutics, and Philae Pharmaceuticals. Remuneration for these activities is paid to LUMC. LUMC also received speaker honoraria from PTC Therapeutics and BioMarin Pharmaceuticals and funding for contract research from Italpharmaco and Alpha Anomeric. D.R.C. is a consultant for Sarepta Therapeutics.

References

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    1. Aartsma-Rus A and Krieg AM (2017). FDA approves Eteplirsen for Duchenne muscular dystrophy: the next chapter in the Eteplirsen Saga. Nucleic Acid Ther 27:1–3 - PMC - PubMed
    1. Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, et al. (2018). Eteplirsen treatment for Duchenne muscular dystrophy: exon skipping and dystrophin production. Neurology 90:e2146–e2154 - PubMed
    1. Aartsma-Rus A and Arechavala-Gomeza V (2018). Why dystrophin quantification is key in the eteplirsen saga. Nat Rev Neurol 14:454–456 - PubMed

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