Journal article

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

Edward C Smith, Laurie S Conklin, Eric P Hoffman, Paula R Clemens, Jean K Mah, Richard S Finkel, Michela Guglieri, Mar Tulinius, Yoram Nevo, Monique M Ryan, Richard Webster, Diana Castro, Nancy L Kuntz, Laurie Kerchner, Lauren P Morgenroth, Adrienne Arrieta, Maya Shimony, Mark Jaros, Phil Shale, Heather Gordish-Dressman Show all

PLoS Medicine | PUBLIC LIBRARY SCIENCE | Published : 2020


BACKGROUND: Treatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti-inflammatory investigational drug that has shown evidence of efficacy in DMD after 24 weeks of treatment at 2.0 or 6.0 mg/kg/day. Here, open-label efficacy and safety experience of vamorolone was evaluated over a period of 18 months in trial participants with DMD. METHODS AND FINDINGS: A multicenter, open-label, 24-week trial (VBP15-003) with a 24-month long-term extension (VBP15-LTE) was conducted by the Cooperative International Neuromuscular..

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Awarded by National Institutes of Health

Awarded by European Commission

Funding Acknowledgements

This work was funded by: National Institutes of Health NINDS R44NS095423 [EPH, PRC] Institutes of Health NICHD 5U54HD090254 [EPH, LSC] Institutes of Health NIAMS U34AR068616 [PRC], Commission Horizons 2020 grant agreement number 667078 [MG], funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.