A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

A Study of Respiratory Muscle Strength, Including Effort-Independent Measures, in Subjects With Late-Onset Pompe Disease

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 .

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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