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

Neuromuscular Disorders

Volumn 20, Issue 5, 2010, Pages 355-362

  Muntoni, F ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; AVI 4658; DYSTROPHIN; OLIGONUCLEOTIDE PHOSPHOROTHIOATE; PRO 051; UNCLASSIFIED DRUG;

ARTICLE; CLINICAL TRIAL; DISEASE COURSE; DRUG ACTIVITY; DRUG EFFICACY; DRUG SOLUBILITY; DUCHENNE MUSCULAR DYSTROPHY; EUROPE; EXON; EXON SKIPPING; GENE MUTATION; HEALTH CARE COST; HUMAN; INNATE IMMUNITY; MOBILIZATION; MUSCLE FUNCTION; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; WORKSHOP;

BIOLOGICAL THERAPY; EUROPE; GENE THERAPY; GUIDELINES AS TOPIC; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; OUTCOME ASSESSMENT (HEALTH CARE);

EID: 77952009340     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2010.03.005     Document Type: Article

References (23)

1. Aartsma-Rus A., Fokkema I., Verschuuren J., et al. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mut 2009, 30:293-299.
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 R.T., et al. 3.03 6-Minute walk test in Duchenne muscular dystrophy: longitudinal observations. Neuromusc Dis 2009, 19:511-674.
5. Florence J., Abresch R.T., 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 R.T., 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 J.E., Florence J.M., Mayhew T.P., et al. Reliable surrogate outcome measures in multi-center clinical trials of Duchenne muscular dystrophy. Muscle Nerve 2007, 35:36-42.
8. Hiller L.B., Wade C.K. Upper extremity functional assessment sales in children with Duchenne muscular dystrophy: a comparison. Arch Phys Med Rehab 1992, 73:527-534.
9. Wagner M.B., Vignos P.J., Carlozzi C., Hull A.L. Assessment of hand function in Duchenne muscular dystrophy. Arch Phys Med Rehab 1993, 74:801-804.
10. Phillips M.F.R., Quinlivan C.M., Edwards R.H.T., Calverley P.M.A. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med 2001, 64:2191-2194.
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 G.J. Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA 2007, 13:1609-1624.
13. van Ommen G.J., 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 J.C., Fokkema I.F., van Ommen G.J., den Dunnen J.T. 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.
16. Levin AA, Yu RZ, Geary RS. Basic principles of pharmacokinetics of antisense oligonucleotide drugs. In: Crooke ST, editor. Antisense drug technology: principles, strategies and applications. 2nd ed. Boca Raton: Taylor and Francis; 2007.
17. Kwoh JT. An overview of the clinical safety experience of first and second generation antisense oligonucleotides. In: Crooke ST, editor. Antisense drug technology. 2nd ed. Boca Raton: Taylor and Francis; 2007.
18. Levin A.A. Biochim Biophys Acta 1999, 1489:69-84.
19. Monteith D.K., Horner M.J., Gillett N.A., et al. Toxicol Pathol 1999, 27:307-317.
20. Iversen PL. Morpholinos. In: Crooke ST, editor. Antisense drug technology. New York: Marcel Dekker, Inc; 2007.
21. van Deutekom J.C., Janson A.A., Ginjaar I.B., et al. Local dystrophin restoration with antisense oligonucleotie PRO051. N Engl J Med 2007, 357:2677-2686.
22. Kinali M, Arechavala-Gomeza V, Feng L et al. Lancet neurology local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in DMD: a single-blind, placebo-controlled, dose-escalation proof-of-concept study. 2009;8:918-928.
23. Hagger L., Woods S. Children and research: a risk of double jeopardy. Int J Children's Rights 2005, 13:51-72.