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Lysosomal Storage Diseases clinical trials at University of California Health

2 research studies open to eligible people

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

    open to eligible people ages 18 years and up

    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.

    at UC Irvine

  • Stem Cell Gene Therapy for Cystinosis

    open to eligible people ages 18 years and up

    This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04)

    at UCSD