Serum biomarkers associated with baseline clinical severity in young steroid-naive Duchenne muscular dystrophy boys
Serum biomarkers associated with baseline clinical severity in young steroid-naive Duchenne muscular dystrophy boys
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DOI：
10.1093/hmg/ddaa132
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发表时间：
2020-08-01
影响因子：
3.5
通讯作者：
Finkel, Richard S.
中科院分区：
文献类型：
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作者：
Dang, Utkarsh J.;Ziemba, Michael;Finkel, Richard S.
Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin in muscle, and while all patients share the primary gene and biochemical defect, there is considerable patient-patient variability in clinical symptoms. We sought to develop multivariate models of serum protein biomarkers that explained observed variation, using functional outcome measures as proxies for severity. Serum samples from 39 steroid-naive DMD boys 4 to
