STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Inclusion Criteria

• •1. Subject has a diagnosis of Pompe disease based on documented deficiency of GAA activity and a documented GAA mutation.
• •2. Male and female subjects between 18 years and 75 years, inclusive and ≥ 50 kg.
• •3. Subject must be currently receiving standard-of-care ERT (alglucosidase alfa) at a dose of 20 mg/kg dose every other week.
• •4. Subject must have been on ERT for the preceding 2 years or more.
• •5. Subject must have an upright forced vital capacity (FVC) within 35 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6 minute walk distance (6MWD) is \> 200 m. Subject must have an upright FVC within 40 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6MWD is ≤ 200 m. If FVC is between 80 and 90% of predicted normal, the subject may enter the study if the percent predicted FVC value drops by 10% predicted or more in supine position
• •6. Subject is able to walk at least 100 m in the 6MWT and the assessment is noted as valid.