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Updated: May 17, 2026

Assessing Functional Performance in the Mdx Mouse Model
Published on: March 27, 2014
Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy
Eric P Hoffman1, Erica Reeves, Jesse Damsker
1Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA. ehoffman@cnmcresearch.org
Abstract:
Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen. Ideally an optimized regimen would maximize efficacy while minimizing side-effect profiles. A new trial, FOR-DMD, aims to address this gap in knowledge. In parallel, there has been progress in the area of "dissociative steroids," drugs that are able to better separate efficacy and side effects, providing a broader therapeutic window.
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