The FOR DMD study will compare three ways of giving corticosteroids to boys with DMD to determine which increases muscle strength the most, and which causes the fewest side effects.
Corticosteroids are currently the only medicine that has been shown to stabilise or even increase muscle strength in boys with DMD over a limited period of time. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems, although the response may vary from boy to boy.
However corticosteroids also cause several side effects. Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug and currently children in different countries and in different neuromuscular centres are prescribed with different corticosteroid, and some are not prescribed with corticosteroids at all.
The FOR DMD study will compare
- daily prednisone (0.75 mg/kg/day)
- intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
- daily deflazacort (0.9 mg/kg/day).
All three dosages are commonly used in boys with Duchenne muscular dystrophy and have shown to be beneficial.
Using the results of this study, we aim to provide patients and families with clearer information about the best way to take these drugs.
The study will be conducted in accordance with the highest standards of care currently available for the management of DMD and of the steroid-related side effects.
Child information sheet (age 4-5)