Cyclosporine Has No Effect in Duchenne Muscular Dystrophy

In a randomized controlled trial, cyclosporine had no effect on muscle strength, function, or quality of life in ambulatory boys with DMD.

Cyclosporine is an immunosuppressive agent initially evaluated for treatment of Duchenne muscular dystrophy (DMD) in boys because of the observed benefits of corticosteroids, which also exert an immunosuppressive effect. Adverse effects limit the use (and therefore efficacy) of corticosteroids in these patients. Cyclosporine showed promise in two nonrandomized studies (Muscle Nerve 1997; 20:469 and Neurology 1993; 43:527) and in the mdx mouse model of DMD (Am J Pathol 2005; 166:477). Now, researchers report the results of a randomized, placebo-controlled trial of cyclosporine (3.5–4.0 mg/kg/day) in 153 boys with DMD. Boys older than 5 years who were still able to walk independently were randomized to receive either cyclosporine or placebo f…

Reviewing Author

Michael Benatar, MD, MS, PhD

Disclosures

Grant / Research supportNIH NeuroBioBank; ALS Association; NIH/National Institute of Neurological Disorders and Stroke; NIH/National Center for Advancing Translational Sciences; FDA; Department of Defense

Editorial boardsCochrane Collaboration

Leadership positions in professional societiesMuscle Study Group Executive Committee

Michael Benatar, MD, MS, PhD

Disclosures

Editorial boardsCochrane Collaboration

Leadership positions in professional societiesMuscle Study Group Executive Committee