Enzyme replacement therapy for infantile-onset pompe disease: curse or cure?
Willems
Jef
J
Paediatric Intensive Care Unit, Great Ormond Street Hospital NHS Trust, London, UK.
Petros
Andy
A
Brierley
Joe
J
D000367:Age Factors; D018450:Disease Progression; D017809:Fatal Outcome; D005260:Female; D005087:Glucan 1,4-alpha-Glucosidase / Q000008:administration & dosage* / Q000009:adverse effects* / Q000172:deficiency; D006003:Glycogen / Q000378:metabolism; D006009:Glycogen Storage Disease Type II / Q000201:enzymology / Q000503:physiopathology / Q000628:therapy*; D006801:Humans; D007231:Infant, Newborn; D008247:Lysosomes / Q000187:drug effects / Q000378:metabolism; D008297:Male; D018908:Muscle Weakness / Q000188:drug therapy / Q000201:enzymology / Q000503:physiopathology; D012121:Respiration, Artificial / Q000009:adverse effects*; D012131:Respiratory Insufficiency / Q000188:drug therapy / Q000201:enzymology / Q000503:physiopathology; D012132:Respiratory Muscles / Q000187:drug effects* / Q000201:enzymology / Q000503:physiopathology; D017211:Treatment Failure; D015300:Ventilator Weaning / Q000706:statistics & numerical data*
D006003:Glycogen; D005087:Glucan 1,4-alpha-Glucosidase