[Enzyme Replacement Therapy for Pompe Disease: The Long-Term Efficacy and Limitation].
The long-term efficacy of enzyme replacement therapy (ERT) in patients with Pompe disease has been unraveled. Cardiac muscle responds well to ERT, whereas motor and respiratory functions do not. The screening of newborns, which enables the early initiation of ERT, has improved outcomes of infantile Pompe patients. Myopathies still exist even in patients in whom ERT is initiated early, and have become recognized as an emerging new phenotype of IP on ERT. In addition, this shows the limitation of currently available ERT. It is expected that the development of therapy will improve the outcome of Pompe disease.
Pediatrics, Hamamatsu Medical University.
Tokiko
T
Fukuda
Hideo
H
Sugie
D000818:Animals; D001343:Autophagy / Q000276:immunology*; D004195:Disease Models, Animal; D056947:Enzyme Replacement Therapy; D006009:Glycogen Storage Disease Type II / Q000276:immunology / Q000628:therapy*; D006801:Humans; D009136:Muscular Dystrophies / Q000473:pathology / Q000628:therapy; D013995:Time