Apply to trial NCT04639336

A few quick questions so the study team can decide if you might be a fit.

Recruiting

Cognitive and Neurological Pathologies in Pompe Disease

The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. The investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We forward your profile to the study team

    They see only the answers needed to decide if you can be screened.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

We save your progress under this email so you can come back later, and the study team uses it to reach you.

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