Long-term corticosteroid treatment delays respiratory decline and heart disease in boys with Duchenne muscular dystrophy (DMD) regardless of daily or intermittent use, a real-world study reported. The study, “Cardiorespiratory Progression Over 5 Years and Role of Corticosteroids in Duchenne Muscular Dystrophy: A Single-Site…

Catabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (DMD), the small molecule edasalonexent preserved bone health, whereas the corticosteroid prednisolone caused bone loss. An ongoing double-blind and randomized Phase 3 trial (NCT03703882), called PolarisDMD, is evaluating…

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial…

Boys with Duchenne muscular dystrophy (DMD) on a long-term daily regimen of glucocorticoids are more likely to develop spinal fractures, have poorer growth and gain more body fat, than do those on intermittent use, a two-year study reports. But study findings also suggest that those…

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials. The new findings follow a reassessment of pooled data obtained in the placebo group…

Deflazacort delays loss of ambulation (LOA) in Duchenne muscular dystrophy (DMD) patients by an average of 3.8 years more than standard of care corticosteroid treatments prednisone/prednisolone, post-hoc analyses of a Phase 3 clinical trial found. The new conclusions follow a reassessment of data obtained in the placebo group of the completed ACT DMD clinical trial (NCT01826487). The goal…