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Fabry Disease clinical trials at UCSD

2 in progress, 1 open to eligible people

Showing trials for
  • An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

    open to eligible people ages 18 years and up

    This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease.

    La Jolla, California and other locations

  • Extension Study of 1 mg/kg Pegunigalsidase Alfa in Patients With Fabry Disease

    Sorry, accepting new patients by invitation only

    The objective of PB-102-F60 is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

    La Jolla, California and other locations

Our lead scientists for Fabry Disease research studies include .

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