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Glycogen Storage Disease Type II clinical trials at UCSF
1 in progress, 1 open to new patients

  • Pompe Disease Registry

    The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: - To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease; - To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; - To characterize and describe the Pompe disease population as a whole; and - To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme®.

    Sacramento, California and other locations