Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys

Autor: Cinrg Vamorolone, Michael Ziemba, Yetrib Hathout, Eric P. Hoffman, Laurie S. Conklin, Paula R. Clemens, Investigators, Utkarsh J. Dang
Rok vydání: 2020
Předmět:
Zdroj: Human Molecular Genetics
ISSN: 1460-2083
Popis: 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-naïve DMD boys 4 to
Databáze: OpenAIRE