Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state., MATSUOKA Takashi;MIWA Yoshiyuki;TAJIKA Makiko;SAWADA Madoka;FUJIMAKI Koichiro;SOGA Takashi;TOMITA Hideshi;UEMURA Shigeru;NISHINO Ichizo;FUKUDA Tokiko;SUGIE Hideo;KOSUGA Motomichi;OKUYAMA Torayuki;UMEDA Yoh, Mol Genet Metab Rep,
9:98 - 105, Nov. 2016,
Peer-reviewed,
DOI:10.1016/j.ymgmr.2016.11.001