Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease. [electronic resource]
- Neuromuscular disorders : NMD Jun 2008
- 447-52 p. digital
Publication Type: Case Reports; Journal Article; Research Support, Non-U.S. Gov't
0960-8966
10.1016/j.nmd.2008.04.009 doi
Adolescent Adult Animals CHO Cells--drug effects Child Cricetinae Cricetulus Female Glycogen Storage Disease Type II--drug therapy Humans Longitudinal Studies Male Muscle, Skeletal--drug effects Treatment Outcome alpha-Glucosidases--therapeutic use