The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy.

ABSTRACT:

Background

Significant advances in the development of SMN-restoring therapeutics have occurred since 2010 when very effective biological treatments were reported in mouse models of spinal muscular atrophy. As these treatments are applied in human clinical trials, there is pressing need to define quantitative assessments of disease progression, treatment stratification, and therapeutic efficacy. The electrophysiological measures Compound Muscle Action Potential and Motor Unit Number Estimation are reliable measures of nerve function. In both the SMN∆7 mouse and a pig model of spinal muscular atrophy, early SMN restoration results in preservation of electrophysiological measures. Currently, clinical trials are underway in patients at post-symptomatic stages of disease progression. In

SUBMITTER: Arnold W 

PROVIDER: S-EPMC4724465 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature