reply to post by lindsay1984
I came across your post while searching for information on DMD. My wife and I were just given the same diagnosis for our six year old. We're waiting
on genetic test results to confirm, but the reality of this type of diagnosis is crushing. I've spent the last few weeks scouring the depths of the
internet to get a better understanding of what DMD is, and what treatment options are available. I'm not a doctor, but I have enough of a background
in chemistry and biology to understand some of the more technical information available. I wanted to touch base here and share some of the information
I've pieced together.
First, I wanted to touch on your statements regarding steroid use in DMD. It should be made clear that the only currently medically accepted treatment
for DMD is glucocortico-steroids (prednisone if you are in the US). There is a substantive difference between gc and anabolic steroids used by
athletes and weight lifters, not the least of which being that they are administered orally, not via injection. The side effects have a significantly
different profile, lower risk for creating the kind of organ damage that anabolic steroids cause, for example.
That said, I'm not completely sold on the use of gc steroids in the ambulatory phase of DMD. Science hasn't been able to fully evaluate the
mechanism by which prednisone affects DMD symptoms, and there isn't consensus on dosing schedules or when best to begin therapy. That said, it's
probably the most fully researched option, and in our litigation-happy climate I don't fault doctors for being reluctant to skip the established
treatment and go to alternative options. We haven't been to see the doctors at our local MDA clinic yet, but I am sadly expecting a similar level of
push towards prednisone.
The expected side effects from treatment that most concern me would include weight gain (seems contraindicated in a population with degenerative
muscle conditions), osteoporosis, and immunosuppression (also seems contraindicated in a population that suffers from high incidences of respiratory
illnesses). The flip side is that it has a clinically established result of maintaining ambulation for a few years beyond the mean, and reduces
scoliosis in non-ambulatory kids.
So the primary goal of steroid therapy is to maintain muscle mass and prolong ambulation, and help treat cardiomyopathy and scoliosis in kids with
There are alternative medications that have been evaluated for use off-label (not for their intended purpose) that show promise in some of the same
areas. Oxandrolone, which funny enough actually *is* an anabolic steroid, has been used since the mid-80's to treat patients who suffer from
degenerative, wasting illnesses (en.wikipedia.org...
) . AFAIK, it's approved for use with patients who have HIV, Turner's
Syndrome, and hepatitis. Its side effect profile is less severe than prednisone, and in clinical trials to test it in kids with DMD, I think the most
serious side effect was nausea. It has shown measurable improvement in lean muscle mass and measurable strength, but less than what has been shown in
glucocortico steroid treatment. IMO, it's worth considering as a first line treatment, prior to loss of ambulation and before prednisone treatment
begins, but I haven't talked to a doctor yet so I don't know why it's not a preferred treatment.
Another option is Insulin-like growth factor (IGF-1) (en.wikipedia.org...
) . I believe it is currently in
clinical trials for treatment of DMD, ALS, and myotonic muscular dystrophy, but it's already been approved for use in growth related disorders. It
has a relatively short list of possible side effects, the most serious being hypoglycemia and possible intracranial pressure (though I don't know
what risk factors might trigger either). It is actually available as a dietary supplement as well as a prescription drug, and from what I've read it
can be absorbed sublingually with a close approximation of intravenous effectiveness (80ish percent compared to IV). That said, the stuff you buy as a
dietary supplement isn't closely regulated, so you can't really know for sure that what you buy off the shelf actually *is* IGF-1, or that it has
been prepared in a manner that would make it available to the body when given sublingually.
There's also flavocoxid (en.wikipedia.org...
), brand name limbrel, which is an anti-inflammatory that is used in osteoarthritis.
It belongs to a class of chemical called flavenoids, which are derived from botanical sources.
Another currently available treatment option is a combination of Isosorbide Dinitrate and Ibuprofen, which when used together has shown clinically
significant improvements, but the combination is still in trials (phase II or III).
Will continue and post more links below.