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Expert Opinion on Orphan Drugs Volume 4, 2016 - Issue 2
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Review

Antisense oligonucleotide development for the treatment of muscular dystrophies

Tri Le BaoCentre for Comparative Genomics, Murdoch University, Perth, Western Australia, Australia
,
Rakesh N. VeeduCentre for Comparative Genomics, Murdoch University, Perth, Western Australia, Australia;Western Australian Neuroscience Research Institute, Perth, Western Australia, Australia
,
Sue FletcherCentre for Comparative Genomics, Murdoch University, Perth, Western Australia, Australia;Western Australian Neuroscience Research Institute, Perth, Western Australia, Australia
&
Steve D. WiltonCentre for Comparative Genomics, Murdoch University, Perth, Western Australia, Australia;Western Australian Neuroscience Research Institute, Perth, Western Australia, AustraliaCorrespondence[email protected]
Pages 139-152 | Received 25 Sep 2015, Accepted 17 Nov 2015, Published online: 15 Dec 2015

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•• Discussion on exon-skipping therapy for DMD.

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• Guidelines for in vivo experiments using exon-skipping antisense oligonucleotides for DMD.

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•• Guidelines for in vitro and in vivo experiments using exon-skipping antisense oligonucleotides for DMD.

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•• Clinical trials on DMD.

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•• Clinical trials on DMD.

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•• Clinical trials on DMD.

  • Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol. 2014;13(10):987–996.
  • Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne’s muscular dystrophy. N Engl J Med. 2011;364(16):1513–1522.
  • Wilton SD, Veedu RN, Fletcher S. The emperor’s new dystrophin: finding sense in the noise. Trends Mol Med. 2015;21(7):417–426.

• Essential discussion on DMD.

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