Article

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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Langue : ANGLAIS