AB126. Enzyme replacement therapy in patient with mucopolysaccharidosis type I: a case report

BACKGROUND AND OBJECTIVE: Mucopolysaccharidosis I (MPS I) is a rare, recessively inherited, lysosomal storage disorder caused by deficiency on the enzyme α-L-iduronidase. This defect results in accumulation of heparan and dermatan sulfate in different tissues and organs due to a deficiency in the ca...

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Bibliografische gegevens
Gepubliceerd in:Ann Transl Med
Hoofdauteurs: Can, Ngoc Thi Bich, Vu, Dung Chi, Le, Hang Thi Thuy, Nguyen, Khanh Ngoc, Bui, Huong Thi
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: AME Publishing Company 2015
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Part 4: Oral/poster
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4563421/
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3978/j.issn.2305-5839.2015.AB126
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