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โ† Trials
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Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Recruitingn/aAges any - anyAllDuke University
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In plain words

What the trial is, and whether it could suit you

This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease * Must provide a written informed consent
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What it tests

  • Observational
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Where

1 site
Duke University Medical Center
Durham, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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