Nicolino, Marc <br/><br/>
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.  [electronic resource] 

 -  Genetics in medicine : official journal of the American College of Medical Genetics  Mar 2009 
 - 210-9 p.  digital 
<br/><br/> Publication Type: Clinical Trial; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1530-0366 
<br/><br/><label>Standard No.: </label>10.1097/GIM.0b013e31819d0996  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Body Height<br/>Body Weight<br/>Child, Preschool<br/>Cough--chemically induced<br/>Echocardiography<br/>Enzyme-Linked Immunosorbent Assay<br/>Female<br/>Glycogen--metabolism<br/>Glycogen Storage Disease Type II--drug therapy<br/>Humans<br/>Immunoglobulin G--blood<br/>Infant<br/>Kaplan-Meier Estimate<br/>Male<br/>Muscle, Skeletal--drug effects<br/>Skin Diseases--chemically induced<br/>Time Factors<br/>Treatment Outcome<br/>alpha-Glucosidases--adverse effects