Effect of enzyme therapy in juvenile patients with Pompe disease: a three-year open-label study.

van Capelle, C I

Effect of enzyme therapy in juvenile patients with Pompe disease: a three-year open-label study. [electronic resource] - Neuromuscular disorders : NMD Dec 2010 - 775-82 p. digital

Publication Type: Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't

1873-2364

10.1016/j.nmd.2010.07.277 doi


Adolescent
Child
Child, Preschool
Enzyme Replacement Therapy
Female
Glucan 1,4-alpha-Glucosidase--therapeutic use
Glycogen Storage Disease Type II--physiopathology
Humans
Male
Muscle, Skeletal--physiopathology
Recombinant Proteins--therapeutic use
Treatment Outcome