Publications

A new tool for tracking the progression of spinal muscular atrophy (SMA): quantitative magnetic resonance imaging (qMRI).

24/08/2017
Description:

In a Roche collaboration study with the University of Basel Children’s Hospital in Switzerland, researchers examined ways of tracking change in people with Type 3 SMA over a 1-year period using different biomarkers.  They also looked at whether these biomarkers correlated with well established measures of mobility and movement (MFM [motor function measure] and 6MWT [6minute walk test]).

Download Publication

Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients

03/06/2017
Description:

Authors: F. Rouault, V. Christie-Brown, R. Broekgaarden, N. Gusset, D. Henderson, P. Marczuk, I. Schwersenz, G. Bellis, C. Cottet .

Journal: Neuromuscular Disorders
DOI: 10.1016/j.nmd.2017.01.018

Standards of Care in spinal muscular atrophy

03/06/2017
Description:

Audience: Patient Advocacy Organizations, Europe+

Download Publication