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  • SPK-GAA-100


    Enrollment Information

    Call (602) 406-1466 or E-Mail [email protected]

    The Gregory W. Fulton Neuromuscular Center

    Learn More

    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


    ALS, Pompe disease

    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.