28
Participants
Start Date
October 31, 2015
Primary Completion Date
July 31, 2019
Study Completion Date
July 31, 2019
History and physical
Subjects will undergo a physical exam and medical history review, including review of genetic test results.
Tidal breathing
Subjects' breathing patterns will be evaluated at rest and at reduced/eliminated ventilator support.
Maximal respiratory pressures
Subjects' strongest inspiratory and expiratory pressures will be measured.
Peak cough flow
Subjects' strongest coughs will be measured.
Pediatric Evaluation of Disability Inventory
Assesses the functional capabilities of children with disabilities.
PedsQL Multidimensional Fatigue Scale
Assesses general fatigue, sleep/rest fatigue, and cognitive fatigue.
Review of ventilation requirements
Subjects's use of mechanical ventilation reviewed by the study team.
University of Florida, Gainesville
Collaborators (1)
Audentes Therapeutics
INDUSTRY
University of Florida
OTHER