Neuromuscular Disorders
Volume 20, Issue 5 , Pages 355-362 , May 2010

The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: Report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009

  • F. Muntoni

      Affiliations

    • Corresponding Author InformationTel.: +44 207 79052608; fax: +44 207 9052832.
    • ,
  • on behalf of the meeting steering committee, and of the TREAT-NMD Network

Received 20 January 2010

References 

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  2. Bushby K, Finkel R, Birnkrant DJ et al. For the DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1; diagnosis, and pharmacological and psychosocial management and part 2: implementation of multidisciplinary care. Lancet Neurol 2009; Nov 27, e-Print.
  3. McDonald C, Coleman K, Henricson E, et al. 3.04 Assessment of StepWatchTM activity monitoring in a phase 2b study of Ataluren (PTC123TM) in nonsense mutation DMD/BMD. Neuromus Dis. 2009;19:511–674
  4. McDonald C, Henricson E, Abresch RT, et al. 3.03 6-Minute walk test in Duchenne muscular dystrophy: longitudinal observations. Neuromusc Dis. 2009;19:511–674
  5. Florence J, Abresch RT, Eagle M, et al. Reproducibility and correlation of pre-treatment outcome measures in a phase 2b study of Ataluren (PTC123TM) in nonsense mutation Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2009;19:511–674
  6. Eagle M, Abresch RT, Florence J. Use of a novel system for grading timed Function test performance in phase 2b study of Ataluren (PTC124TM) in nonsense mutation Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2009;19:511–674
  7. Mayhew JE, Florence JM, Mayhew TP, et al. Reliable surrogate outcome measures in multi-center clinical trials of Duchenne muscular dystrophy. Muscle Nerve. 2007;35:36–42
  8. Hiller LB, Wade CK. Upper extremity functional assessment sales in children with Duchenne muscular dystrophy: a comparison. Arch Phys Med Rehab. 1992;73:527–534
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  11. Iannaccone ST, Hynan LS, Morton A, Buchanan R, Limbers CA, Varni JW. The PedsQL in pediatric patients with Spinal Muscular Atrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Generic Core Scales and Neuromuscular Module. Neuromuscul Disord 2009;19:805-812. (AmSMART Group).
  12. Aartsma-Rus A, van Ommen GJ. Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA. 2007;13:1609–1624
  13. van Ommen GJ, van Deutekom J, Aartsma-Rus A. The therapeutic potential of antisense-mediated exon skipping. Curr Opin Mol Ther. 2008;10:140–149
  14. Aartsma-Rus A, van Deutekom JC, Fokkema IF, van Ommen GJ, den Dunnen JT. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve. 2006;34:135–144
  15. Henry SP, Kim T-W, Kramer-Stickland K, Zanardi TA, Fey RA, Levin AA. Chapter 12: toxicologic Properties of 2- O -methoxyethyl chimeric antisense inhibitors in animals and man. in antisense drug technology; principals, strategies and applications. Stanley T Crooke, editor. (Isis Pharmaceuticals, Inc). Carlsbad California, New York:Marcel Dekker Inc., 2001.
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PII: S0960-8966(10)00110-0

doi: 10.1016/j.nmd.2010.03.005

Neuromuscular Disorders
Volume 20, Issue 5 , Pages 355-362 , May 2010

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