Cognitive and Neurological Pathologies in Pompe Disease

Status: Recruiting
Location: See location...
Study Type: Observational
SUMMARY

In this study, the investigators will collect clinical information as well as complete the following procedures to assess central nervous system (CNS) and peripheral nervous system (PNS) involvement: neuroimaging with MRI, MRS and DTI; quantitative muscle ultrasound, small fiber neuropathy screening list (SFNSL); cognition and developmental assessments including an audiological exam; physical therapy and speech evaluation. These assessments will be collected once a year for up to six years.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: No
View:

• Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)

Locations
United States
North Carolina
Duke University Medical Center
Recruiting
Durham
Contact Information
Primary
Gretchen Nichting
gretchen.nichting@duke.edu
+1 919 660 0757
Time Frame
Start Date: August 3, 2016
Estimated Completion Date: August 2024
Participants
Target number of participants: 45
Treatments
Patients with Pompe disease
Related Therapeutic Areas
Sponsors
Leads: Duke University

This content was sourced from clinicaltrials.gov

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