Enzyme replacement therapy in mucopolysaccharidosis type I: Progress and emerging difficulties

Journal of Inherited Metabolic Disease

Volumn 24, Issue 2, 2001, Pages 245-250

  Wraith, J E ^a  

^a ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

DERMATAN SULFATE; GLYCOSAMINOGLYCAN; HEPARAN; LEVO IDURONIDASE; LYSOSOME ENZYME; RECOMBINANT ENZYME;

ANIMAL EXPERIMENT; ANIMAL MODEL; AORTA VALVE DISEASE; ARTICLE; BONE MARROW TRANSPLANTATION; CARDIOMYOPATHY; CARDIOPULMONARY INSUFFICIENCY; CAUSE OF DEATH; CLINICAL ARTICLE; CLINICAL FEATURE; CLINICAL TRIAL; CORNEA DISEASE; DEATH; DISEASE ASSOCIATION; DISEASE MODEL; DISEASE SEVERITY; DOG; DRUG INFUSION; ENZYME ACTIVITY; ENZYME REPLACEMENT; FACE DEFORMITY; FATALITY; GLYCOSAMINOGLYCAN METABOLISM; HEART DISEASE; HUMAN; HURLER SYNDROME; IMMUNOLOGY; INTELLIGENCE; JOINT STIFFNESS; LEARNING DISORDER; LIFESPAN; LIVER SIZE; LYSOSOME STORAGE DISEASE; NONHUMAN; PATIENT SELECTION; PHENOTYPE; RESPIRATORY DISTRESS; REVIEW; RISK ASSESSMENT; SCHEIE SYNDROME; SKELETON MALFORMATION; TREATMENT OUTCOME;

ANIMALS; BONE MARROW TRANSPLANTATION; DOGS; HUMANS; IDURONIDASE; MUCOPOLYSACCHARIDOSIS I;

EID: 0035000479     PISSN: 01418955     EISSN: None     Source Type: Journal    
DOI: 10.1023/A:1010379320378     Document Type: Article

References (9)

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3. Kakkis ED, Muenzer J, Tiller G, et al (1998) Recombinant α-L-iduronidase replacement therapy in mucopolysaccharidosis I: results of a human clinical trial. Annual Meeting of the American Society for Human Genetics, 1998, Denver, abstract 128.
4. Kakkis ED, Muenzer J, Tiller G, et al (2000) Enzyme replacement therapy in mucopolysaccharidosis I: current status of patients up to 104 weeks of therapy (Abstract). ISIT MPS, Joint meeting of International Symposium on Innovative Therapies and 6th International Symposium on Mucopolysaccharidosis and Related Diseases, Minneapolis.
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