Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings

Molecular Genetics and Metabolism

Volumn 109, Issue 3, 2013, Pages 315-316

  Laraway, Sarah ^a   Breen, Catherine ^a   Mercer, Jean ^a   Jones, Simon ^a   Wraith, James E ^a  

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

Author keywords

Enzyme replacement therapy; Mucopolysaccharidosis

Indexed keywords

GLYCOSAMINOGLYCAN; LARONIDASE;

AORTA VALVE REGURGITATION; ARTICLE; CASE REPORT; CHILD; CLINICAL FEATURE; CORNEA LESION; DISEASE COURSE; ECHOCARDIOGRAPHY; ENZYME REPLACEMENT; FEMALE; FORCED VITAL CAPACITY; FROZEN SHOULDER; HEART VALVE; HIP; HUMAN; HURLER SYNDROME; MALE; MEDICAL HISTORY; MISSENSE MUTATION; MITRAL VALVE PROLAPSE; MITRAL VALVE REGURGITATION; MOTOR ACTIVITY; MOTOR DYSFUNCTION; MUSCLE RIGIDITY; ORTHOPEDIC SURGERY; PRESCHOOL CHILD; PRIORITY JOURNAL; RANGE OF MOTION; SHOULDER; SIBLING; VALVULAR HEART DISEASE;

EID: 84891371607     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.04.023     Document Type: Article

References (8)

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