Lead Sponsor

Astellas Gene Therapies

Age

16 - 69 years

Sex

ALL

Healthy Volunteers

No

•Participant has a documented clinical diagnosis of LOPD.
•Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]).
•Participant is willing and able to comply with study visits and procedures.
•Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study.

•Participant previously received an AAV-related product (any serotype).
•Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
•Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility).
•Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility).