Lysosomal Storage Diseases clinical trials at University of California Health

2 in progress, 0 open to eligible people

Showing trials for

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

    Sorry, in progress, not accepting new patients

    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

    Sorry, in progress, not accepting new patients

    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 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

    at UCSD

Our lead scientists for Lysosomal Storage Diseases research studies include .

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