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Glycogen Storage Disease Type II clinical trials at UC Irvine

1 in progress, 0 open to eligible people

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  • A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

    Sorry, currently not accepting new patients, but might later

    The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

    Orange, California

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