Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease characterized by progressive muscle weakness, multiple system involvement and premature mortality. Effective treatments for DMD through clinical trials and natural history studies are currently underway. Clinical trials in DMD typically include several outcome measures of motor function. Research sites and studies have been found to have slightly different operational definitions for a given functional outcome resulting in different procedures and protocols for these measurements.

Objective

The goal of this study is to establish agreement among experts in the field around best practices in collecting functional outcome data in DMD providing researchers and clinicians with guidance on best pra

SUBMITTER: Senesac CR 

PROVIDER: S-EPMC6698889 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature