Published in
Elsevier, Molecular Genetics and Metabolism Reports, (9), p. 98-105, 2016
DOI: 10.1016/j.ymgmr.2016.11.001
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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
Journal article published in 2016 by
Takashi Matsuoka,
Yoshiyuki Miwa,
Makiko Tajika 
,
Madoka Sawada,
Koichiro Fujimaki,
Takashi Soga,
Hideshi Tomita ,
Shigeru Uemura,
Ichizo Nishino ,
Tokiko Fukuda,
Hideo Sugie,
Motomichi Kosuga,
Torayuki Okuyama,
Yoh Umeda
,
Madoka Sawada,
Koichiro Fujimaki,
Takashi Soga,
Hideshi Tomita ,
Shigeru Uemura,
Ichizo Nishino ,
Tokiko Fukuda,
Hideo Sugie,
Motomichi Kosuga,
Torayuki Okuyama,
Yoh Umeda
This paper is made freely available by the publisher.
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