What do I think about the clinical trial?

There have been some studies testing Deflazacort in Duchenne MD and
comparing it to Prednisone in particular. The general conclusion
seems to be:

1) Deflazacort is beneficial in slowing the progression of Duchenne.

2) Its benefit is comparable to that of Prednisone.

3) It causes less weight gain than Prednisone (one of the major side
effects of Prednisone).

There hasn't ever been a clinical trial of prednisone in
dysferlinopathy, but quite a few individual patients have been put on
it by their doctors, either because they were misdiagnosed with
polymyositis, or because it was thought that prednisone might help
other forms of MD besides Duchenne. There have been a few references
to these "experiments" in some of the literature articles, and in no
case has Prednisone been reported to be beneficial.

I'm not sure what the rationale is for thinking Deflazacort would be
beneficial in dysferlinopathy if its effect is similar to Prednisone
in DMD and prednisone doesn't help in dysferlinopathy. (Maybe there is
a good reason that I don't know)

Anyway, it's good that someone is organizing a clinical trial, and
maybe the result will surprise me!

Brad