Study was terminated early due to a company decision not based on safety concerns

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

Fifteen subjects were planned for enrollment; there were no screening failures; 8 subjects were enrolled. Two subjects were enrolled from 701-301 and the remaining 6 subjects enrolled directly into 701-201 (no parent study)

28 weeks

The study was planned to have 15 participants. However, the study was terminated prematurely. Only 8 participants were enrolled. Seven participants had last assessment at Days 146, 168, 172, 174, 179, 188, 196, translating to one participant at Week 20 and six participants Weeks 24-28.