Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes

This collaborative study out of Taiwan compared the impact of very early (by 10 days of age) to early (by 1 month of age) initiation of enzyme replacement therapy on long-term health outcomes in patients with infantile onset Pompe disease (IOPD), a lysosomal storage disorder. The authors conclude that starting enzyme replacement therapy just a few days earlier may improve long-term outcomes for IOPD patients.

Authors: Chia-Feng Yang, Chen Chang Yang, Hsuan-Chieh Liao, Ling-Yi Huang, Chuan-Chi Chiang, Hui-Chen Ho, Chih-Jou Lai, Tzu-Hung Chu, Tsui-Feng Yang, Ting-Rong Hsu, Wen-Jue Soong, Dau-Ming Niu

The Journal of Pediatrics 2016; 169: 174-80.