CNMC0302: AN OPEN-LABEL PILOT STUDY OF PENTOXYFILLINE IN STEROID NAIVE DUCHENNE

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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 the most common lethal inherited disorder worldwide. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. Not only because the uniform use of prednisone (the only drug proven to be beneficial) is hampered by potential adverse effects, but also because it may very well be the case that ultimately a combination of both gene and drug therapy will be needed to cure Duchenne children. The Cooperative International Neuromuscular Research Group is a recently established international collaborative clinical network, focused on developing rational therapies for neuromuscular disorders of childhood. The purpose of this study is to evaluate the effect of pentoxifylline on muscle strength in children with DMD in a multicenter open-label pilot study.