Health and economic outcomes of newborn screening for infantile-onset Pompe disease
John S. Richardson, Alex R. Kemper, Scott D. Grosse, Wendy K. K. Lam, Angela M. Rose, Ayesha Ahmad, Achamyeleh Gebremariam, Lisa A. Prosser
Topics & Concepts
Newborn screeningMedicineEnzyme replacement therapyPediatricsDiseaseCohortMicrosimulationQuality-adjusted life yearHealth careCost effectivenessInternal medicineEngineeringEconomicsRisk analysis (engineering)Transport engineeringEconomic growthLysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusMetabolism and Genetic Disorders