Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial
Inclusion body myositis is most frequent in patients older than 50 years. In this pilot study, we aimed to test the efficacy of sirolimus in patients with the disease. The study was a randomized, double-blind, placebo-controlled, proof-of-concept, phase 2b trial. Canker sores were the most frequent side-effect and were mainly mild or moderate in ten patients.