Late-onset Pompe disease (LOPD) causes progressive muscle weakness and respiratory insufficiency in children and adults; current treatment is enzyme replacement therapy (ERT). Two surveys were conducted to better understand the burdens and needs from the perspective of the patient (pt) and clinician. US-based adult pts with LOPD were invited through pt associations; the survey was administered by a trained professional. Clinicians (MDs) with expertise in LOPD completed their survey online. Both surveys collected information on demographics, disease, treatment, and effect on daily living and employment.

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