Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

Genetics in Medicine

Volumn 11, Issue 3, 2009, Pages 210-219

  Nicolino, Marc ^a   Byrne, Barry ^b   Wraith, J E ^c   Leslie, Nancy ^d   Mandel, Hanna ^e   Freyer, David R ^f   Arnold, Georgianne L ^g   Pivnick, Eniko K ^h   Ottinger, C J ⁱ   Robinson, Peter H ^j   Loo, John Charles A ^k   Smitka, Martin ^l   Jardine, Philip ^m   Tatò, Luciano ⁿ   Chabrol, Brigitte ^o   McCandless, Shawn ^p   Kimura, Shigemi ^q   Mehta, L ^r   Bali, Deeksha ^s   Skrinar, Alison ^t   Morgan, Claire ^t   Rangachari, Lakshmi ^t   Corzo, Deya ^u   Kishnani, Priya S ^s,v   more..

^a UNIVERSITÉ DE LYON   (France)

^b UNIVERSITY OF FLORIDA   (United States)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^d CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^e RAMBAM MEDICAL CENTER   (Israel)

^f HELEN DEVOS CHILDREN S HOSPITAL   (United States)

^g UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^h UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

ⁱ Fort Wayne Neurological Center   (United States)

^j ROYAL HOSPITAL FOR SICK CHILDREN   (United Kingdom)

^k LONG BEACH MEMORIAL MEDICAL CENTER   (United States)

^l DRESDEN UNIVERSITY OF TECHNOLOGY   (Germany)

^m BRISTOL ROYAL HOSPITAL FOR CHILDREN   (United Kingdom)

ⁿ UNIVERSITY OF VERONA   (Italy)

^o TIMONE HOSPITAL   (France)

^p UNIVERSITY HOSPITALS OF CLEVELAND   (United States)

^q KUMAMOTO UNIVERSITY   (Japan)

^r MOUNT SINAI MEDICAL CENTER   (United States)

^s DUKE UNIVERSITY   (United States)

^t GENZYME CORPORATION   (United States)

^u MILLENNIUM PHARMACEUTICALS INC   (United States)

^v DUKE UNIVERSITY MEDICAL CENTER   (United States)

more..

Author keywords

Acid maltase deficiency; Cardiomyopathy; Enzyme replacement therapy; Glycogen storage disease type II; Lysosomal acid glu cosidase; Motor development; Myozyme, alglucosidase alfa; Pompe disease; Recombinant human GAA

Indexed keywords

ALPHA GLUCOSIDASE; GLUCAN 1,4 ALPHA GLUCOSIDASE; IMMUNOGLOBULIN G ANTIBODY; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE;

ARTICLE; ASSISTED VENTILATION; CHILD; CLINICAL ARTICLE; CLINICAL EVALUATION; CLINICAL TRIAL; COHORT ANALYSIS; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; COUGHING; DEATH; DISEASE FREE SURVIVAL; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SAFETY; ECHOCARDIOGRAPHY; ENZYME ACTIVITY; FEMALE; FEVER; FLUSHING; GLYCOGEN STORAGE DISEASE TYPE 2; HEART LEFT VENTRICLE MASS; HUMAN; HYPERTENSION; HYPOXIA; INFANT; LONG TERM CARE; MALE; MOTOR PERFORMANCE; MULTICENTER STUDY; OPEN STUDY; PRESCHOOL CHILD; RISK FACTOR; SIDE EFFECT; SKIN DISEASE; SUBCUTANEOUS FAT DISORDER; TACHYCARDIA; TACHYPNEA; TREATMENT OUTCOME; URTICARIA; VENTILATOR; WALKING;

ALPHA-GLUCOSIDASES; BODY HEIGHT; BODY WEIGHT; CHILD, PRESCHOOL; COUGH; ECHOCARDIOGRAPHY; ENZYME-LINKED IMMUNOSORBENT ASSAY; FEMALE; GLYCOGEN; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNOGLOBULIN G; INFANT; KAPLAN-MEIERS ESTIMATE; MALE; MUSCLE, SKELETAL; SKIN DISEASES; TIME FACTORS; TREATMENT OUTCOME;

EID: 63449127241     PISSN: 10983600     EISSN: None     Source Type: Journal    
DOI: 10.1097/GIM.0b013e31819d0996     Document Type: Article

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