Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will test respiratory muscle strength initially and again after 24 weeks in subjects treated or not treated with BMN 701 .
Study Type
OBSERVATIONAL
Enrollment
8
University of Florida
Gainesville, Florida, United States
Royal Brompton Hospital
London, United Kingdom
Maximal Inspiratory Pressure (MIP)
Pulmonary measure of change in maximal inspiratory pressure (MIP)
Time frame: Baseline, Week 28
Maximal Expiratory Pressure (MEP)
Pulmonary measure of change in maximal expiratory pressure (MEP)
Time frame: Baseline, Week 28
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