Treatment of sporadic inclusion body myositis with bimagrumab.

ABSTRACT:

Objective

To study activin signaling and its blockade in sporadic inclusion body myositis (sIBM) through translational studies and a randomized controlled trial.

Methods

We measured transforming growth factor β signaling by SMAD2/3 phosphorylation in muscle biopsies of 50 patients with neuromuscular disease (17 with sIBM). We tested inhibition of activin receptors IIA and IIB (ActRII) in 14 patients with sIBM using one dose of bimagrumab (n = 11) or placebo (n = 3). The primary outcome was the change in right thigh muscle volume by MRI at 8 weeks. Lean body mass, strength, and function were secondary outcomes. Twelve of the patients (10 bimagrumab, 2 placebo) participated in a subsequent 16-week observation phase.

Results

Muscle SMAD2/3 phosphorylation was higher in s

SUBMITTER: Amato AA 

PROVIDER: S-EPMC4277670 | biostudies-literature | 2014 Dec

REPOSITORIES: biostudies-literature