Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training

Status: Completed
Location: See location...
Study Type: Observational

The primary purpose of this study is to: Document the developmental outcomes of individuals with Pompe disease treated with long-term enzyme-replacement therapy (ERT) through school-age (ages 6-18) using measures of cognitive functioning, academic skills, and speech and language abilities. Investigate possible cognitive processing speed weaknesses using BrainBaseline neurocognitive assessment software. Investigate the relationship between behavior and other developmental factors including speech and language ability and cognitive ability. Explore if the use of selected iPad applications may help strengthen cognitive processing speed in children with Pompe disease

Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 18
Healthy Volunteers: No

• Age range 6-18 years

• Diagnosis of classic Pompe disease by enzyme or molecular methods

• Patient, parent, or legal guardian is willing and able to give written informed consent

• English speaking child and care giver.

United States
North Carolina
Duke University Medical Center
Time Frame
Start Date: December 2015
Completion Date: March 3, 2021
Target number of participants: 22
Related Therapeutic Areas
Leads: Duke University

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