DAILY VS WEEKLY PREDNISONE THERAPY IN DUCHENNE MUSCULAR DYSTROPHY

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Duchenne muscular dystrophy (DMD) is among the most common of genetic disorders worldwide. Yet, to date, the only clinical treatments that have been shown to improve the health and mobility of DMD patients are the corticosteroids prednisone and deflazacort. Both agents shoe immune modulatory function, and both decreased the invasion of T-cells in DMD muscle. However there is growing evidence that additional pathophysiological pathways are responsible, in part, for the efficacy of corticosteroids. Including improvement of calcium homeostasis, inhibition of muscle protein degradation, and improvement of energy metabolism. While many physicians consider chronic steroid administration as "standard of care" in DMD, only about half of physicians and patient families in the USA opt to use steroids due to the side effects associated with its chronic use. Many of those that use steroid, delay therapy until the disease is advanced because of concern that risks outweigh the benefits of therapy. Some alternate regimes use for other autoimmune diseases have been found to be ineffective in DMD; however, large dose steroid given intermittently, which have been found to be effective and have fewer side effects in other autoimmune disorders, have not been evaluated in DMD in adequately powered controlled trials. This study will evaluate the high dose prednisone adminsitered on an intermittent schedule in pediatric patients with DMD. Finding an alternate dose regimen that would be at least as effective as daily therapy, but has fever side effects is the goal of this proposal. If successful, this regimen would likely become a standard of care in DMD and permit onset of therapy earlier, which might be even more beneficial.