Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
- 1. John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust and Newcastle University, Newcastle, United Kingdom
- 2. University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania
- 3. Neuromuscular Centre, Department of Pediatric Neurology, Motol University Hospital, 2nd Medical School, Charles University, Prague, Czech Republic
Description
Question For steroidal anti-inflammatory drugs, can efficacy be retained while safety concerns are reduced among boys with Duchenne muscular dystrophy (DMD) with the novel partial receptor agonist vamorolone?
Findings A randomized, double-blind, placebo- and prednisone-controlled trial of vamorolone (2 dose groups) was carried out in 121 patients with DMD. The trial met the primary (time to stand velocity after 24 weeks for vamorolone, 6 mg/kg per day vs placebo) and first 4 sequential secondary motor function end points; vamorolone showed loss of bone morbidities compared with prednisone, with no stunting of growth and no deleterious changes in bone biomarkers.
Meaning This study found that vamorolone, a dissociative steroidal anti-inflammatory, was able to reduce bone morbidities while retaining efficacy.
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Efficacy and safety of vamorolone vs placebo and prednisone.pdf
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