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. 2017 Feb;27(1):1-3.
doi: 10.1089/nat.2016.0657. Epub 2016 Dec 8.

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Annemieke Aartsma-Rus  1 , Arthur M Krieg  2
Affiliations

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Annemieke Aartsma-Rus et al. Nucleic Acid Ther. 2017 Feb.
No abstract available

Keywords: oligonucleotide; regulatory; splicing modulation; therapy.

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Conflict of interest statement

A.A.R. report being employed by LUMC, which has patents on exon skipping technology. As a coinventor of some of these patents, A.A.R. may receive a share of potential royalties. A.A.R. reports being an ad hoc consultant for BioMarin, PTC, BMS, and Summit. Remuneration for these activities and speaker honoraria for BioMarin and PTC-organized satellite symposia go to LUMC. A.M.K. was previously employed as Sarepta's CSO and has been a consultant to companies working on RNA and DMD therapeutics.

References

    1. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm
    1. Goemans NM, Tulinius M, van den Hauwe M, Kroksmark AK, Buyse G, Wilson RJ, van Deutekom JC, de Kimpe SJ, Lourbakos A, and Campion G. (2016). Long-term efficacy, safety, and pharmacokinetics of drisapersen in Duchenne muscular dystrophy: results from an open-label extension study. PLoS One 11:e0161955. - PMC - PubMed
    1. http://investors.bmrn.com/releasedetail.cfm?ReleaseID=973536
    1. Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, et al. ; Eteplirsen Study Group. (2013). Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 74:637–647 - PubMed
    1. www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drug...