The natural history of MPS I: global perspectives from the MPS I Registry

Genetics in Medicine

Volumn 16, Issue 10, 2014, Pages 759-765

  Beck, Michael ^a   Arn, Pamela ^b   Giugliani, Roberto ^c   Muenzer, Joseph ^d   Okuyama, Torayuki ^e   Taylor, John ^f   Fallet, Shari ^f  

^a JOHANNES GUTENBERG UNIVERSITY   (Germany)

^b NEMOURS CHILDREN S CLINIC   (United States)

^c HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^d UNIVERSITY OF NORTH CAROLINA   (United States)

^e NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT   (Japan)

^f GENZYME CORPORATION   (United States)

Author keywords

MPS I; natural history; registry; symptoms

Indexed keywords

ADOLESCENT; ADULT; ASIA; CHILD; CLINICAL TRIAL; EUROPE; FEMALE; GENETIC ASSOCIATION; GENETICS; GENOTYPE; HUMAN; INFANT; MALE; MUCOPOLYSACCHARIDOSIS I; MULTICENTER STUDY; NORTH AMERICA; ONSET AGE; PHENOTYPE; PRESCHOOL CHILD; PREVALENCE; REGISTER; SOUTH AND CENTRAL AMERICA; STATISTICS AND NUMERICAL DATA;

ADOLESCENT; ADULT; AGE OF ONSET; ASIA; CHILD; CHILD, PRESCHOOL; EUROPE; FEMALE; GENETIC ASSOCIATION STUDIES; GENOTYPE; HUMANS; INFANT; LATIN AMERICA; MALE; MUCOPOLYSACCHARIDOSIS I; NORTH AMERICA; PHENOTYPE; PREVALENCE; REGISTRIES;

EID: 84931420298     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2014.25     Document Type: Article

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