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Scientific Articles

Timed Function Tests Have Withstood the Test of Time as Clinically Meaningful and Responsive Endpoints in Duchenne Muscular Dystrophy

key information

source: Muscle & Nerve

year: 2018

authors: McDonald CM

summary/abstract:

Invesitgators from the ImagingDMD study are to be congratulated for their recent study of longitudinal assessments of common clinical trial endpoints in boys with Duchenne muscular dystrophy (DMD) including the 6-minute walk test (6MWT), and timed function tests (TFTs) such as timed supine to stand, 4-stair climb, and 10 meter run/walk.

Using these tests, Arora and colleagues generated a large data set on 92 corticosteroid treated patients describing the contemporary natural history of DMD in ambulatory patients 5 to 12.9-years of age with up to 4 years of follow-up in a subset of patients. These TFTs were first utilized as clinical endpoints in Duchenne muscular dystrophy by the Clinical Investigation in Duchenne Dystrophy (CIDD) Study Group in 1981 and these endpoints were shown to be responsive to corticosteroid treatment effects in young DMD subjects in 1989. Since that time TFTs have been commonly employed primary and secondary endpoints in ambulatory DMD clinical trials. 

organization: University of California, USA

DOI: 10.1002/mus.26334

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