New treatments are urgently needed to slow disease progression in patients with DMD
Currently, glucocorticoids are the only available medical treatment that can slow the decline in muscle strength and function, irrespective of the disease-causing mutation. 3,5,13,17,18 However, the effect is only partial and clinical use is limited by well-known side effects caused by steroids. 3,5,13,17,18 A recent study showed that up to 42% of DMD patients aged 10 years and older had either never used steroids or have discontinued their use. 5 Loss of respiratory function with increasing age continues to be a major cause of morbidity and mortality in patients with DMD, and new treatments to support respiratory function are needed urgently. 2,6,13,17
If you do decide that you want your son to use steroids, then there will be some extra checks that are necessary to make sure that they are having the desired effect and not causing any problems. It will be very important that you attend these appointments. Your son will need to have some blood and urine tests to check everything is OK to use the steroids, and to check that he is immune to chicken pox (if not he will need to be immunised against it as chicken pox can be very serious in children who are on steroids). He will also need a scan of his bones and an eye check (these will need to be repeated at intervals while he is on the steroids), and a good assessment of how he is getting on at the moment.