Disease progression modeling of the North Star Ambulatory Assessment for Duchenne Muscular Dystrophy.

ABSTRACT: Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by decreased or absent dystrophin gene leading to progressive muscle degeneration and weakness in young boys. Disease progression models for the North Star Ambulatory Assessment (NSAA), a functional measurement widely used to assess outcomes in clinical trials, were developed using a longitudinal population modeling approach. The relationship between NSAA total score over time, loss of ambulation, and potential covariates that may influence disease progression were evaluated. Data included individual participant observations from an internal placebo-controlled phase II clinical trial and from the external natural history database for male patients with DMD obtained through the Cooperative International Neuromuscular Resear

SUBMITTER: Hibma JE 

PROVIDER: S-EPMC10014057 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature