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Gene therapy for pompe disease effective in mice, poised for human trials – Medical Xpress


Medical Xpress

Gene therapy for pompe disease effective in mice, poised for human trials
Medical Xpress
After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients.

2017-01-26 NEWS
About Michael Zand

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