Glucocorticoids can improve strength and preserve ambulation in Duchenne, but are accompanied with unwanted side-effects. Recent preclinical data in Duchenne models showed spironolactone, a commonly used medication, increases muscle membrane stabilization with reduced negative side-effects associated with steroids.
A Phase 1 trial is actively recruiting participants.
This program is sponsored by Nationwide Children’s Hospital.
|Spironolactone Versus Prednisolone in DMD|