Effect of different corticosteroid dosing regimens on clinical outcomes in boys with Duchenne muscular dystrophy: a randomized clinical trial

M Guglieri, K Bushby, MP McDermott, KA Hart, R Tawil… - Jama, 2022 - jamanetwork.com
Importance Corticosteroids improve strength and function in boys with Duchenne muscular
dystrophy. However, there is uncertainty regarding the optimum regimen and dosage.
Objective To compare efficacy and adverse effects of the 3 most frequently prescribed
corticosteroid regimens in boys with Duchenne muscular dystrophy. Design, Setting, and
Participants Double-blind, parallel-group randomized clinical trial including 196 boys aged 4
to 7 years with Duchenne muscular dystrophy who had not previously been treated with …