CNMC0705: A DOUBLE-BLINDED RANDOMIZED PLACEBO CONTROLLED STUDY OF DAILY

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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 a progressive disease of skeletal muscle caused by the absence of dystrophin due to a genetic mutation in the X-linked dystrophin gene. The absence of dystrophin results in a fragile muscle membrane that permits an abnormal permeability to electrolytes, especially calcium. The increase in intracellular calcium triggers a pathological cascade of events that ultimately resultsin muscle necrosis and fibrosis which impedes normal muscle regeneration. The increase knowledge of the pathophysiology of DMD opens the opportunity for pharmacological treatment, with the purpose of altering the disease processs and or reverting the muscle degeneration. Pentoxifylline (PTX) is a potent anti-inflammatory and anti-fibrotic compound that produced 51% increase in strength in exercised mdx mice. Its efficacy in DMD is not proven. However preliminary studies in DMD and other muscular dystrophyies are encouraging. DMD children treated with steroid who are weak but stable or deteriorating despite all proven treatments may benefit from the addition of PTX treatment.