The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) subjects receiving standard-of-care enzyme replacement therapy (ERT). Additionally, information gained may be used in the design and conduct of future studies in LOPD subjects.
The objective of this study is to evaluate the baseline characteristics and degree of change over time in clinical outcome measures commonly used to evaluate patients with LOPD.
Study Type
OBSERVATIONAL
Enrollment
12
University of California, Irvine
Irvine, California, United States
Evaluate degree of change in muscle function and respiratory endpoints over time
To evaluate the degree of change in muscle function and respiratory endpoints over time in patients with Late Onset Pompe disease
Time frame: 6-15 month
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