Abstract
The lack of natural history data and well-characterized outcome measures limits the ability to conduct therapeutic clinical trials in Duchenne Muscular Dystrophy (DMD). This study describes pulmonary function tests (PFTs) from 341 participants with confirmed DMD from the Cooperative International longitudinal study. The objective of this study is to describe the pulmonary function characteristics of individuals with DMD and to determine whether steroid therapy is associated with improvements in respiratory function. Participants in this study ranged from 6–28 years of age from 20 centers and 10 countries. PFT measures included forced vital capacity (FVC, %FVC), forced expiratory volume in 1 s (FEV1, %FEV1), peak expiratory flow rate (PEFR, %PEFR), maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP), and peak cough flow (PCF). The PFT measures were evaluated by current corticosteroid use, age groups ( 15), and ambulatory status (ambulatory, part-time wheelchair user, full-time wheelchair user). All PFT measures except PCF were significantly different in the 3 age groups (p
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