A Study of Repiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
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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 .
Condition or disease
Late-onset Pompe Disease
Procedure: Respiratory muscle strength measurements by different techniques.
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures
Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study entry
Willing and able to comply with all study procedures
Requires ventilatory support while awake and in the upright position
Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise patient well-being, study completion, or data collection
Allergy to tools or procedures used for respiratory muscle testing
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases