作者
Jerry R Mendell, Louise R Rodino‐Klapac, Zarife Sahenk, Kandice Roush, Loren Bird, Linda P Lowes, Lindsay Alfano, Ann Maria Gomez, Sarah Lewis, Janaiah Kota, Vinod Malik, Kim Shontz, Christopher M Walker, Kevin M Flanigan, Marco Corridore, John R Kean, Hugh D Allen, Chris Shilling, Kathleen R Melia, Peter Sazani, Jay B Saoud, Edward M Kaye, Eteplirsen Study Group
发表日期
2013/11
期刊
Annals of neurology
卷号
74
期号
5
页码范围
637-647
简介
Objective
In prior open‐label studies, eteplirsen, a phosphorodiamidate morpholino oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with genetic mutations amenable to skipping exon 51. The present study used a double‐blind placebo‐controlled protocol to test eteplirsen's ability to induce dystrophin production and improve distance walked on the 6‐minute walk test (6MWT).
Methods
DMD boys aged 7 to 13 years, with confirmed deletions correctable by skipping exon 51 and ability to walk 200 to 400 m on 6 MWT, were randomized to weekly intravenous infusions of 30 or 50 mg/kg/wk eteplirsen or placebo for 24 weeks (n = 4/group). Placebo patients switched to 30 or 50 mg/kg eteplirsen (n = 2/group) at week 25; treatment was open label thereafter. All patients had muscle biopsies at baseline and week 48. Efficacy included dystrophin‐positive fibers and distance walked …
学术搜索中的文章
JR Mendell, LR Rodino‐Klapac, Z Sahenk, K Roush… - Annals of neurology, 2013