To skip or not to skip: That is the question for duchenne muscular dystrophy

Molecular Therapy

Volumn 21, Issue 12, 2013, Pages 2131-2132

  Wood, Matthew J A ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

DRISAPERSEN; ETEPLIRSEN; PLACEBO;

DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; DRUG TREATMENT FAILURE; DUCHENNE MUSCULAR DYSTROPHY; EDITORIAL; EXON SKIPPING; HUMAN; RISK BENEFIT ANALYSIS; TREATMENT OUTCOME;

CLINICAL TRIALS AS TOPIC; GENETIC THERAPY; HUMANS; MALE; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES; UTROPHIN;

EID: 84890109756     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.252     Document Type: Editorial

References (5)

1. Fairclough, RJ, Wood, MJ and Davies, KE (2013). Therapy for Duchenne muscular dystrophy: Renewed optimism from genetic approaches. Nat Rev Genet 14: 373-378
2. Goemans, NM, Tulinius, M, van den Akker, JT, Burm, BE, Ekhart, PF, Heuvelmans, N et al. (2011). Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 364: 1513-1522
3. Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K et al. (2011). 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 378: 595-605
4. Lourbakos, A, Beekman, C, Holling, T, Testerink, J, Duinsbergen, D, Giannakopoulos S et al. (2013). P.11.21 Bioanalysis of a double blind, placebo-controlled clinical phase 2 study of drisapersen for the treatment of boys suffering from Duchenne muscular dystrophy and comparison to clinical outcome results. Neuromuscul Disord 23: 805
5. McDonald, CM, Henricson, EK, Abresch, RT, Florence, JM, Eagle, M, Gappmaier, E et al. (2013). The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve 48: 343-356