In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe’s Disease

Amazing therapeutic success led by UCSF Pediatrics joined by Duke UniversityOttawa University, and Washington University via in-utero enzyme-replacement therapy for infantile-onset Pompe’s disease: Patients with early-onset lysosomal storage diseases are ideal candidates for prenatal therapy because organ damage starts in utero. After receiving in-utero ERT and standard postnatal therapy, the current patient had normal cardiac and age-appropriate motor function postnatally, was meeting developmental milestones, had normal biomarker levels, and was feeding and growing well at 13 months of age.

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