Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II

Mucopolysaccharidosis I and II are lysosomal storage disorders that, despite treatment with hematopoietic cell transplantation (HCT) and/or enzyme replacement therapy (ERT), continue to cause significant skeletal abnormalities leading to pain, stiffness, physical dysfunction, and short stature. Tumo...

詳細記述

保存先:
書誌詳細
出版年:Mol Genet Metab Rep
主要な著者: Polgreen, Lynda E., Kunin-Batson, Alicia, Rudser, Kyle, Vehe, Richard K., Utz, Jeanine J., Whitley, Chester B., Dickson, Patricia
フォーマット: Artigo
言語:Inglês
出版事項: Elsevier 2017
主題:
Research Paper
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC5238608/
https://ncbi.nlm.nih.gov/pubmed/28119823
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.01.002
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