Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.

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.

• Authors: Eric P Hoffman, Erica Reeves, Jesse Damsker, Kanneboyina Nagaraju, John M McCall, Edward M Connor, Kate Bushby
• Journal: Physical medicine and rehabilitation clinics of North America (Phys Med Rehabil Clin N Am) Vol. 23 Issue 4 Pg. 821-8 (Nov 2012) ISSN: 1558-1381 [Electronic] United States
• PMID: 23137739 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review)
• Copyright: Copyright © 2012 Elsevier Inc. All rights reserved.

Chemical References

• Glucocorticoids
• Pregnenediones
• deflazacort
• Prednisone

Topics

• Drug Discovery
• Glucocorticoids (chemistry, pharmacology, therapeutic use)
• Humans
• Muscular Dystrophy, Duchenne (drug therapy)
• Prednisone (therapeutic use)
• Pregnenediones (therapeutic use)