OBJECTIVE: To characterize the natural progression of infantile-onset Pompe disease. STUDY DESIGN: Retrospective chart reviews of 168 patients with documented acid alpha-glucosidase deficiency and symptom onset by 12 months of age; Kaplan-Meier…
BACKGROUND: Specialized tertiary care facilities developed in response to the increasing numbers of adults with congenital heart disease (CHD). Because this patient population comprises a relatively new area of specialized cardiovascular interest,…