TY - JOUR
T1 - The gross motor function measure™ is a valid and sensitive outcome measure for spinal muscular atrophy
AU - Nelson, Leslie
AU - Owens, Hollis
AU - Hynan, Linda S.
AU - Iannaccone, Susan T.
AU - AmSMART Group, Group
N1 - Copyright:
Copyright 2008 Elsevier B.V., All rights reserved.
PY - 2006/6
Y1 - 2006/6
N2 - Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure™ is a measure of motor function. We showed previously that the Gross Motor Function Measure™ is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure™ when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure™ total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P<0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure™ is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy.
AB - Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure™ is a measure of motor function. We showed previously that the Gross Motor Function Measure™ is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure™ when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure™ total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P<0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure™ is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy.
KW - Clinical trials
KW - Gross motor function measure
KW - Outcome measures
KW - Quantitative muscle testing
KW - Spinal muscular atrophy
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U2 - 10.1016/j.nmd.2006.03.005
DO - 10.1016/j.nmd.2006.03.005
M3 - Article
C2 - 16632361
AN - SCOPUS:33745027696
SN - 0960-8966
VL - 16
SP - 374
EP - 380
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 6
ER -