Steroids are now 'gold standard' in Duchenne. While many physicians recommend daily regimens, there are still others who suggest alternate regimens. Data from the recent CINRG trial comparing weekend dosing to daily dosing suggest nearly equal benefit. But to be fair, the trial is not following the boys long-term to understand if the age of loss of ambulation varies from daily to weekend dosing.
And while steroids do offer considerable benefit, there is also considerable risk. Sadly, just a few weeks ago, our community suffered a major blow. We all mourn the loss of a 7 year old boy in the UK. He died from an undiagnosed peptic ulcer. He was on steroids. While we do not have the post mortem results, there is something in our hearts that connects the two – steroids and peptic ulcers. And we worry. While this may be an isolated incident, it needs to be a warning sign as well.
Boys with Duchenne need experts to care for them. While many patients are seen in clinics far from home, it is essential that the Duchenne expert is communicating with your primary care physician. Duchenne is complex and your son is likely to be the only child with Duchenne in his/her practice. As parents, we are the advocates for our sons and often that means we are the ones to insist on clear communication and immediate access to our son's Duchenne physicians in case of emergency. If your son complains of pain or if you see something that is 'not exactly right,' seek help immediately.
Please take a moment to read Nick Catlin of Action Duchenne's blog: