Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
University of Florida
With
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Subjects with Pompe disease who are scheduled to receive a NeuRx Diaphragm pacer (DPS) will be invited to participate in this study. The following tests will be performed during participation in the study:
1. Forced expiratory tests
2. Maximal Inspiratory Pressure (MIP)
3. Resting Breathing Pattern
4. Phrenic Nerve Function Evaluation with EMG
5. Severe Respiratory Insufficiency Questionnaire
6. Magnetic stimulation
7. Diaphragm conditioning consisting of:
1. The external stimulator settings
2. The duration of diaphragm stimulation
3. Off-ventilator spontaneous breathing (SB)
Age
2–65
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Diagnosis of Pompe disease as defined by mutational analysis, GAA enzyme activity assay in blood spot, and/or fibroblast culture less than 40% of control value
Medically and functionally eligible for NeuRx DPS implantation, as determined by a clinical surgical evaluation. The surgeon will determine eligibility through pulmonary function tests, ability to stimulate your diaphragm, and arterial blood gas levels. Eligibility may also be determined by your response to traditional respiratory strengthening exercises without a pacemaker.
History of mechanical ventilation dependence (six or more hours of invasive or non-invasive mechanical ventilation support daily for at least 21 days in duration
You may not be if
Current participation in another treatment study (other than enzyme replacement therapy) directed at improving diaphragm function
