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Newborn Screening + Enzyme Replacement Therapy =Improved Lysosomal Storage Disorder: Outcomes in Infantile-Onset Pompe Disease

This article discusses the benefits of newborn screening early detection and early intervention therapies for patients with Pompe and other lysosomal storage disorders.

Title: Newborn Screening + Enzyme Replacement Therapy =Improved Lysosomal Storage Disorder: Outcomes in Infantile-Onset Pompe Disease
Author: Hans C. Andersson, MD
Date: January 29, 2015
Link: http://www.jpeds.com/article/S0022-3476(14)01197-4/pdf