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  • SPK-GAA-100

    The purpose of this study is to obtain information pertaining to the occurrence of SPK-GAA antibodies and other laboratory measures in participants with Late Onset Pompe Disease (LOPD) on an enzyme replacement therapy regimen. Additionally, this study will collect retrospective laboratory and medically-relevant historic data relating to Pompe disease from participants in hopes to provide a better understanding of LOPD features to improve the design of a future phase 1/2 interventional gene therapy trial. In most cases, participants will attend a one-time clinic visit, though the option of two separate study visits may be available.

    Principal Investigator: Shafeeq Ladha, MD

    Lysosomal Storage Disorders (LSD) Registry Program

    The Lysosomal Storage Disorders (LSD) Registry Program is a multicenter, international, observational program for people with certain rare diseases. It is designed to track the natural history and outcomes of patients. Currently, patients diagnosed with Pompe disease may participate in this registry program.

    No experimental intervention is given as part of this program. As such, participants will be assessed clinically and receive standard-of-care treatment as determined by their physician. Physicians will determine the actual frequency of necessary assessments according to a patient’s individualized need for medical care and routine follow up.

    Principal Investigator: Shafeeq Ladha, MD

    Enrollment Contact
    Jessie Duncan
    (602) 406-1466
    [email protected]

    About Barrow Neurological Institute
    Since our doors opened as a regional specialty center in 1962, we have grown into one of the premier destinations in the world for neurology and neurosurgery. Our experienced, highly skilled, and comprehensive team of neurological specialists can provide you with a complete spectrum of care–from diagnosis through outpatient neurorehabilitation–under one roof. Barrow Neurological Institute: Discover. Educate. Heal.