CLINICAL TRIAL: SIX-MONTH (26 WEEKS) CLINICAL TRIAL OF GENTAMICIN IN DUCHENNE MD

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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. We hypothesize that gentamicin given over six months will significantly improve strength in DMD patients. Thirteen percent of DMD patients have a stop codon mutation based on our mutational analysis of 141 non-deleted patients.13 To fulfill its potential as a long-term therapeutic agent for DMD, gentamicin must be safe and improve muscle strength. The primary goal of this study will be to determine the safety of gentamicin over six months. The premise for the six-month study proposed here is that the serum CK reduction was an early indicator of membrane stability in previous trials. Therefore, long-term administration will permit amplification of dystrophin expression. Measurable amounts of dystrophin should be detected at the sarcolemma protecting the muscle from necrosis, while at the same time increasing muscle strength.