Serum biomarkers associated with baseline clinical severity in young steroid-naive Duchenne muscular dystrophy boys
- Dang, UJ
- Ziemba, M
- Clemens, PR
- Hathout, Y
- Conklin, LS
- Hoffman, EP
- Smith, EC
- Mah, JK
- Guglieri, M
- Nevo, Y
- Kuntz, N
- McDonald, CM
- Tulinius, M
- Ryan, MM
- Webster, R
- Castro, D
- Finkel, RS
DOIAbstract
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 <7 years enrolled into a clinical trial of vamorolone were studied (NCT02760264). Four assessments of gross motor function were carried out for each participant over a 6-week interval, and their mean was used as response for biomarker models. Weighted correlation network analysis was used for unsupervised clustering ...
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