title: 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.affiliation: Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA. ehoffman@cnmcresearch.org
authors.forename: Eric P
authors.initials: EP
authors.lastname: Hoffman
authors.forename: Erica
authors.initials: E
authors.lastname: Reeves
authors.forename: Jesse
authors.initials: J
authors.lastname: Damsker
authors.forename: Kanneboyina
authors.initials: K
authors.lastname: Nagaraju
authors.forename: John M
authors.initials: JM
authors.lastname: McCall
authors.forename: Edward M
authors.initials: EM
authors.lastname: Connor
authors.forename: Kate
authors.initials: K
authors.lastname: Bushby
mesh_terms: D055808:Drug Discovery; D005938:Glucocorticoids / Q000737:chemistry / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D011241:Prednisone / Q000627:therapeutic use; D011282:Pregnenediones / Q000627:therapeutic use
chemical_list: D005938:Glucocorticoids; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone