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Modeling Clinical Outcome Variability in Duchenne Muscular Dystrophy

November 25, 2021 at 11:00 AM to 12:00 PM

Location:Virtual (Zoom)

CUIDS Distinguished Speaker Series

Modeling Clinical Outcome Variability in Duchenne Muscular Dystrophy
with Dr. Utkarsh Dang

Abstract: Duchenne muscular dystrophy (DMD) is a progressive disease caused by loss of dystrophin in muscle. While all patients share the primary gene and biochemical defect, there is considerable patient–patient variability in clinical symptoms at an early age as well as over time. Our objective was twofold: model early age clinical severity in steroid-naive DMD boys through circulating protein biomarkers, and infer clusters of similarly progressing boys over time. Hence, we developed multivariate models (cross-sectional) of serum protein biomarkers that explained observed variation, using functional outcome measures as proxies for severity. Furthermore, we investigated clustering clinical outcome trajectories via modeling a multivariate combination of early-age clinical outcome measurements simultaneously to better explain disease progression rates. We show that performance of DMD boys was effectively modeled with serum proteins, and that multiple groups of DMD motor trajectories progressing at a unique rate can be established at an early age.

Seminar Moderator:

Dr. Tracey P. Lauriault, Associate Professor, Critical Media and Big Data, School of Journalism and Communication, Carleton University