The drug idebenone produced notably less decline in standard respiratory function measures in patients with Duchenne muscular dystrophy (DMD), according to a study published in The Lancet.
Patients with Duchenne muscular dystrophy who took the oral drug idebenone for 12 months showed significantly less decline in standard respiratory function measures including peak expiratory flow (PEF) in a placebo-controlled, 66-patient trial, researchers said.
PEF as a percentage of predicted normal fell by 3.05 points (95% CI -7.07 to 0.97) during the trial with idebenone, compared with a decrease of 9.01 points (95% CI -13.18 to -4.84, P=0.0001) in the placebo group, reported Gunnar M. Buyse, MD, of University Hospitals Leuven in Belgium, and colleagues.
The difference between groups in the modified intent-to-treat analysis was 5.96 points favoring idebenone (95% CI 0.16 to 11.76), the researchers wrote online in The Lancet. The study is also scheduled to be reported at the American Academy of Neurology annual meeting.