Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.

ABSTRACT:

Objective

This study prospectively assessed putative promising biomarkers for use in assessing infants with spinal muscular atrophy (SMA).

Methods

This prospective, multi-center natural history study targeted the enrollment of SMA infants and healthy control infants less than 6 months of age. Recruitment occurred at 14 centers within the NINDS National Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) Network. Infant motor function scales and putative electrophysiological, protein and molecular biomarkers were assessed at baseline and subsequent visits.

Results

Enrollment began November, 2012 and ended September, 2014 with 26 SMA infants and 27 healthy infants enrolled. Baseline demographic characteristics of the SMA and control infant cohorts aligned

SUBMITTER: Kolb SJ 

PROVIDER: S-EPMC4748311 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature