Title:
IMPROVED COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
Document Type and Number:
WIPO Patent Application WO/2014/144978
Kind Code:
A3
Abstract:
Improved compositions and methods for treating muscular dystrophy by administering eteplirsen, an antisense molecule capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, are described.
Inventors:
KAYE EDWARD M (US)
Application Number:
PCT/US2014/029610
Publication Date:
November 06, 2014
Filing Date:
March 14, 2014
Export Citation:
Assignee:
SAREPTA THERAPEUTICS INC (US)
International Classes:
A61P21/00; A61K31/7125
Other References:
"ETEPLIRSEN - Inhibitor of Dystrophin Expression - Treatment of Duchenne Muscular Dystrophy", DRUGS OF THE FUTURE 38(1), 1 January 2013 (2013-01-01), pages 13 - 17, XP055135338, Retrieved from the Internet [retrieved on 20140819], DOI: 10.1358/dof.2013.38.1.1924780
ED KAYE: "Results of the Eteplirsen Phase 2b and Phase 2b Extension Study in Duchenne Muscular Dystrophy", NUCLEIC ACID THERAPEUTICS, 1 December 2012 (2012-12-01), New Rochelle, pages A13, XP055135355, Retrieved from the Internet [retrieved on 20140819], DOI: 10.1089/nat.2012.1501
MICHELE A SCULLY ET AL: "Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy", EXPERT OPINION ON ORPHAN DRUGS, vol. 1, no. 1, 1 January 2013 (2013-01-01), pages 33 - 46, XP055135324, DOI: 10.1517/21678707.2013.746939
ANONYMOUS: "Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients - Full Text View - ClinicalTrials.gov", 22 January 2013 (2013-01-22), XP055135303, Retrieved from the Internet [retrieved on 20140819]
DUBOWITZ NEUROMUSCULAR ET AL: "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study", LANCET PUBLISHED ONLINE JULY, 1 January 2011 (2011-01-01), pages 595 - 605, XP055135286, Retrieved from the Internet [retrieved on 20140819], DOI: 10.1016/S0140-
JERRY R MENDELL ET AL: "Eteplirsen for the treatment of Duchenne muscular dystrophy", ANNALS OF NEUROLOGY, 1 November 2013 (2013-11-01), United States, pages 637 - 647, XP055135300, Retrieved from the Internet DOI: 10.1002/ana.23982
ED KAYE: "Results of the Eteplirsen Phase 2b and Phase 2b Extension Study in Duchenne Muscular Dystrophy", NUCLEIC ACID THERAPEUTICS, 1 December 2012 (2012-12-01), New Rochelle, pages A13, XP055135355, Retrieved from the Internet
MICHELE A SCULLY ET AL: "Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy", EXPERT OPINION ON ORPHAN DRUGS, vol. 1, no. 1, 1 January 2013 (2013-01-01), pages 33 - 46, XP055135324, DOI: 10.1517/21678707.2013.746939
ANONYMOUS: "Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients - Full Text View - ClinicalTrials.gov", 22 January 2013 (2013-01-22), XP055135303, Retrieved from the Internet
DUBOWITZ NEUROMUSCULAR ET AL: "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study", LANCET PUBLISHED ONLINE JULY, 1 January 2011 (2011-01-01), pages 595 - 605, XP055135286, Retrieved from the Internet
JERRY R MENDELL ET AL: "Eteplirsen for the treatment of Duchenne muscular dystrophy", ANNALS OF NEUROLOGY, 1 November 2013 (2013-11-01), United States, pages 637 - 647, XP055135300, Retrieved from the Internet
Attorney, Agent or Firm:
MANDRAGOURAS, Amy, E. et al. (One Post Office SquareBoston, MA, US)
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