UA-UCD-Sibling concordance for clinical features of Duchenne and Becker muscular dystrophy

2013

Jennifer Andrews
[email protected]
520-626-6816

The correlation of markers of disease severity among brothers with Duchenne and Becker muscular dystrophy has implications for clinical guidance and clinical trials. Sibling pairs with Duchenne and Becker muscular dystrophy were compared for ages when they reached clinical milestones of disease progression, including ceased ambulation, scoliosis of ≥ 20, and development of cardiomyopathy. The median age at which younger brothers reached each milestone, compared to their older brothers ranged from 25 months younger than their older brother for development of cardiomyopathy to 2 months older for ceased ambulation. For each additional month of ambulation by the older brother, the hazard of ceased ambulation by the younger brother decreased by 4%. The ages when siblings reach clinical milestones of disease severity vary widely. However, the time to ceased ambulation for older brothers predicts the time to ceased ambulation for their younger brothers.

Ducheen and Becker Muscular Dystrophy, Ambulation, Cardiomyopathy, Scoliosis, Sibling features

Peer-reviewed publications in scholarly journals submitted

Professionals

N/A