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A closer look at ARSA activity in a patient with metachromatic leukodystrophy

Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease mainly caused by a deficiency of arylsulfatase A activity. The typical clinical course of patients with the late infantile form includes a regression in motor skills with progression to dysphagia, seizures, hypoto...

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書目詳細資料
發表在:Mol Genet Metab Rep
Main Authors: Doherty, Kathleen, Frazier, S. Barron, Clark, Matthew, Childers, Anna, Pruthi, Sumit, Wenger, David A., Duis, Jessica
格式: Artigo
語言:Inglês
出版: Elsevier 2019
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6383325/
https://ncbi.nlm.nih.gov/pubmed/30828547
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2019.100460
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