Clinical Outcome Measures in Spinal Muscular Atrophy
Abstract
Spinal muscular atrophy is one of the most devastating neurological diseases of childhood. Affected infants and children suffer from often severe muscle weakness caused by degeneration of lower motor neurons in the spinal cord and brainstem. Identification of the causative genetic mutation in most cases has resulted in development of potential treatment strategies. To test these new drugs, clinically feasible outcomes are needed. Several...
Paper Details
Title
Clinical Outcome Measures in Spinal Muscular Atrophy
Published Date
Jun 9, 2009
Journal
Volume
24
Issue
8
Pages
968 - 978
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