Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

ABSTRACT:

Objective

To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

Methods

Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly golodirsen; part 2 is an ongoing, open-label evaluation. Safety and pharmacokinetics were primary and secondary objectives of part 1. Primary biological outcome measures of part 2 were blinded exon skipping and dystrophin protein production on muscle biopsies (baseline, week 48) evaluated, respectively, using reverse transcription PCR and Western blot and immunohistochemistry.

Results

Twelve patients were randomized to receive golodirsen (n = 8) or placebo (n = 4) in part 1. All

SUBMITTER: Frank DE 

PROVIDER: S-EPMC7357297 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature