Free Access
Issue
Med Sci (Paris)
Volume 32, Novembre 2016
Les cahiers de myologie
Page(s) 57 - 59
Section Clin d’oeil
DOI https://doi.org/10.1051/medsci/201632s217
Published online 21 November 2016
  1. Kaplan JC. DMD et saut d’exon thérapeutique : tout ce qu’il faut savoir en deux leçons. Les Cahiers de Myologie 2011 ; 5 : 46–47. [Google Scholar]
  2. Kaplan JC. DMD et saut d’exon thérapeutique. Leçon n° 2 : réalisation et perspectives. Les Cahiers de Myologie 2012 ; 6 : 45–46. [Google Scholar]
  3. Aartsma-Rus A, Van Ommen GJB. Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA 2007 ; 13 : 1609–1624. [CrossRef] [PubMed] [Google Scholar]
  4. Van Putten MDWC, Heemskerk H, De Kimpe S, et al. Systemic delivery of antisense oligonucleotides restores dystrophin expression and functionality in the mdx mouse. Nice : Congrès AFM-Myologie, 2000, Poster PW26-322. [Google Scholar]
  5. Bilan de l’European Medicines Agency (EMA) établi en avril 2015. Antisense oligonucleotide-mediated exon skipping therapy development for Duchenne muscular dystrophy (DMD). http://exonskipping.eu/wp-content/uploads/2015/04/Briefing-Document-COST-and-SCOPE-DMD-EMA-meeting-April-2015-FINAL-VERSION.pdf [Google Scholar]
  6. Kaplan JC. L’enfer du génothérapeute est pavé de POC. Med Sci (Paris) 2015 ; 31 (hs3) : 41–44. [CrossRef] [EDP Sciences] [PubMed] [Google Scholar]
  7. Voir l’historique sur le site de Drugs.com : Exondys 51 Approval History. https://www.drugs.com/history/exondys-51.html [Google Scholar]
  8. Voir https://www.drugs.com/newdrugs/fda-approves-exondys-51-eteplirsen-duchenne-muscular-dystrophy-4430.html [Google Scholar]
  9. Suivre la saga Sarepta sur StatNews : FDA panel votes against Sarepta’s drug for Duchenne muscular dystrophy. https://www.statnews.com/pharmalot/2016/04/25/fda-panel-sarepta-muscular-dystrophy/ [Google Scholar]
  10. Senators urge FDA to approve Sarepta drug for Duchenne - STAT https://www.statnews.com/pharmalot/2016/05/24/senators-urge-fda-approve-sarepta-drug-duchenne/ [Google Scholar]
  11. FDA confirms that critic of Sarepta drug has left the agency. https://www.statnews.com/pharmalot/2016/09/14/fda-sarepta-farkas-duchenne. [Google Scholar]
  12. FDA approves Sarepta’s controversial drug for Duchenne muscular dystrophy. https://www.statnews.com/pharmalot/2016/09/19/sarepta-wins-dmd-drug-approval/ [Google Scholar]
  13. Intense FDA bickering. https://www.statnews.com/pharmalot/2016/09/19/sarepta-fda-duchenne-behind-the-decision/ [Google Scholar]
  14. Tough as nails’: Storm swirls around FDA drug cop who approved controversial drug. https://www.statnews.com/2016/09/20/janet-woodcock-sarepta-fda/ [Google Scholar]
  15. Sarepta to charge $300K for Duchenne drug. We tried to be reasonable, CEO says. https://www.statnews.com/pharmalot/2016/09/19/sarepta-duchenne-drug-prices/ [Google Scholar]
  16. Did the FDA set ‘a dangerous precedent’ with its latest drug approval?. https://www.statnews.com/pharmalot/2016/09/19/fda-sarepta-precedent/ [Google Scholar]
  17. Voir le Communiqué de presse de Sarepta du 19/09/2016 : http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=2204492. [Google Scholar]
  18. Voir l’article Eteplirsen sur wikipedia : https://en.wikipedia.org/wiki/Eteplirsen [Google Scholar]

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