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Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam—Part 2: Experience from a nusinersen clinical study

Authors :
Jacqueline Montes
Richard S. Finkel
Kathie M. Bishop
Source :
Muscle & Nerve. 57:142-146
Publication Year :
2017
Publisher :
Wiley, 2017.

Abstract

INTRODUCTION: We examined the feasibility of assessing motor milestone performance of infants with spinal muscular atrophy (SMA) using the Hammersmith Infant Neurological Exam - Part 2 (HINE-2) in a Phase 2 study of nusinersen. METHODS: Nineteen SMA infants were assessed using the HINE-2 at baseline (≤ 7 months of age), and periodically up to 39 months of age. We evaluated whether the HINE-2 was feasible, reliable, and sensitive to change. RESULTS: Motor milestone assessments in SMA infants were feasible using the HINE-2. Baseline test-retest reliability was excellent (R= 0.987; p

Details

ISSN :
10974598 and 0148639X
Volume :
57
Database :
OpenAIRE
Journal :
Muscle & Nerve
Accession number :
edsair.doi.dedup.....549d7805f2c652a848ce6d85668c8cc3
Full Text :
https://doi.org/10.1002/mus.25705