Article
Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.
Human molecular genetics - 29 Aug 2020
Dang Utkarsh J, Ziemba Michael, Clemens Paula R, Hathout Yetrib, Conklin Laurie S, Hoffman Eric P
Abstract excerpt
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...
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