Long-term prognosis of patients with infantile-onset pompe disease diagnosed by newborn screening and treated since birth

Journal of Pediatrics

Volumn 166, Issue 4, 2015, Pages 985-991.e2

  Chien, Yin Hsiu ^a   Lee, Ni Chung ^a   Chen, Chun An ^a   Tsai, Fuu Jen ^b   Tsai, Wen Hui ^c   Shieh, Jeng Yi ^a   Huang, Hsiang Ju ^d   Hsu, Wei Chung ^a   Tsai, Tzu Hsun ^a   Hwu, Wuh Liang ^a  

^a NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

^b CHINA MEDICAL UNIVERSITY HOSPITAL   (Taiwan)

^c CHI MEI MEDICAL CENTER   (Taiwan)

^d Chen Hsin Hospital   (Taiwan)

Author keywords

[No Author keywords available]

Indexed keywords

CREATINE KINASE; IMMUNOGLOBULIN G; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; ALPHA GLUCOSIDASE; GAA PROTEIN, HUMAN; RECOMBINANT PROTEIN;

ANTIBODY TITER; ARTICLE; ARTIFICIAL VENTILATION; CHILD; CLINICAL ARTICLE; COGNITION; COHORT ANALYSIS; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; CYANOSIS; DRUG EFFICACY; ENZYME REPLACEMENT; GLYCOGEN STORAGE DISEASE TYPE 2; HEARING IMPAIRMENT; HEART FUNCTION; HEART LEFT VENTRICLE HYPERTROPHY; HEART LEFT VENTRICLE MASS; HUMAN; INFANT; INFANTILE ONSET POMPE DISEASE; MENTAL DEVELOPMENT; MOTOR DEVELOPMENT; MOTOR PERFORMANCE; MUSCLE WEAKNESS; MYOPIA; NEUROIMAGING; NEWBORN; NEWBORN SCREENING; NUCLEAR MAGNETIC RESONANCE IMAGING; ONSET AGE; OUTCOME ASSESSMENT; POSITIVE END EXPIRATORY PRESSURE; PRESCHOOL CHILD; PRIORITY JOURNAL; PROGNOSIS; PROSPECTIVE STUDY; PTOSIS; RESPIRATORY DISTRESS; SECRETORY OTITIS MEDIA; SURVIVAL; TREATMENT RESPONSE; WHITE MATTER LESION; COMPARATIVE STUDY; FEMALE; FOLLOW UP; GLYCOGEN STORAGE DISEASE TYPE II; INCIDENCE; MALE; PROCEDURES; SURVIVAL RATE; TAIWAN; TIME; TRENDS;

AGE OF ONSET; ALPHA-GLUCOSIDASES; FEMALE; FOLLOW-UP STUDIES; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; INCIDENCE; INFANT; INFANT, NEWBORN; MALE; NEONATAL SCREENING; PROGNOSIS; PROSPECTIVE STUDIES; RECOMBINANT PROTEINS; SURVIVAL RATE; TAIWAN; TIME FACTORS;

EID: 84929637936     PISSN: 00223476     EISSN: 10976833     Source Type: Journal    
DOI: 10.1016/j.jpeds.2014.10.068     Document Type: Article

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