Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Can a Novel Steroidal Anti-Inflammatory Drug Benefit Patients With DMD?

Key clinical point: Oral administration of vamorolone, an investigational steroidal anti-inflammatory drug, may benefit patients with Duchenne muscular dystrophy (DMD), according to an exploratory study.

Major finding: The treatment was safe and well tolerated. Patients who received 2.0 mg/kg per day vamorolone had significantly improved muscle function, as assessed by time to stand, compared with natural history controls.

Study details: An open-label, 24-week extension study of 48 boys with DMD who completed a phase 2a trial.

Disclosures: ReveraGen BioPharma received funds to carry out the study from Actelion Pharmaceuticals, U.S. and European government agencies, and nonprofit foundations. Some of the authors are cofounders of ReveraGen, and others received support from the company.

Citation:

Hoffman EP et al. Neurology. 2019 Aug 26. doi: 10.1212/WNL.0000000000008168.