Enzyme-replacement therapy for Pompe disease

Pediatric Health

Volumn 3, Issue 1, 2009, Pages 41-49

  Pastores, Gregory M ^a   Hughes, Derralynn A ^b  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

Author keywords

Autophagy; Enzyme therapy; Pharmacologic chaperone; Pompe disease; Substrate reduction

Indexed keywords

ADENINE; CYTOSINE; GUANINE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; THYMIDINE;

AGE DISTRIBUTION; CLINICAL TRIAL; DIAPHRAGM DISEASE; DISEASE DURATION; DISEASE SEVERITY; DRUG EFFICACY; DRUG SAFETY; DRUG TARGETING; ENZYME REPLACEMENT; GENOTYPE; GLYCOGEN STORAGE DISEASE TYPE 2; HOMOZYGOSITY; HUMAN; MORTALITY; MUSCLE WEAKNESS; NUCLEIC ACID BASE SUBSTITUTION; RESPIRATORY FAILURE; REVIEW; SURVIVAL RATE; TREATMENT OUTCOME;

EID: 70349649914     PISSN: 17455111     EISSN: None     Source Type: Journal    
DOI: 10.2217/17455111.3.1.41     Document Type: Review

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