Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

ABSTRACT:

Objective

To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three-year progression of eteplirsen-treated patients was compared to matched historical controls (HC).

Methods

Ambulatory DMD patients who were ≥7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open-label basis. The primary functional assessment in this study was the 6-Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used for comparative analysis of 6MWT performance at baseli

SUBMITTER: Mendell JR 

PROVIDER: S-EPMC5064753 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature