Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) exhibits substantial variability in rates of disease progression and response to treatment. This has hindered treatment development and complicated interpretation of drug effects in clinical trials.

Objective

We hypothesized that a multivariate combination of early-age clinical outcome measurements can explain differential disease progression.

Methods

Data on boys with DMD (ages 4-<10 years), both treated with steroidal anti-inflammatories and untreated, were obtained from CINRG Duchenne Natural History Study (n = 209) and vamorolone VBP15-002/003/LTE (n = 46) studies. Velocities from three timed function tests (TFTs; stand from supine, run/walk 10 meters, and climb 4 stairs) were simultaneously modeled in a longitudinal la

SUBMITTER: Fang Y 

PROVIDER: S-EPMC10200136 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature