Michela Guglieri, AAN 2022: Safety and efficacy of corticosteroid regimens for Duchenne muscular dystrophy – Results of an international randomized controlled trial

Corticosteroids can improve muscle function in boys with Duchenne muscular dystrophy and are commonly prescribed for newly diagnosed patients. Dr Michela Guglieri (Newcastle University, Newcastle upon Tyne, United Kingdom) discusses a randomized, double-blind, parallel-group clinical trial investigating comparative safety and efficacy of different corticosteroid regimens for Duchenne muscular dystrophy.

The abstract entitled ‘Comparative Safety and Efficacy of Different Corticosteroid Regimens for Duchenne Muscular Dystrophy: Results of an International Randomized Controlled Trial’ was presented at the American Academy of Neurology (AAN) annual meeting, April 2-7, 2022.

Questions:

1. What were the aims, design and findings of the study you presented at AAN? (0:17)
2. What are the implications of these findings for clinical practice? (2:25)

Disclosures: Michela Guglieri has received grant/research support from EC, NIH, PTC, Sarepta; and has served on the advisory board for Dyne, Pfizer and NS Pharma; and has received honoraria/honorarium from Sarepta.

Support: Interview and filming supported by Touch Medical Media. Interview conducted by Katey Gabrysch.

Filmed as a highlight of AAN 2022