Project to Optimize Testing Measures for Spinal Muscular Atrophy Clinical Tests, Phase I
New York, USA
Gemeinsames Projekt von SMA Foundation (Auftraggeber), Muscular Dystrophy Association, Families of SMA, Fight SMA und SMA Europe e.V.
This study is focused on optimizing currently existing rating scales for Spinal Muscular Atrophy (SMA) in order to ensure effective drug testing in future SMA clinical trials. It is critical for SMA rating scales to be sensitive to changes in patient function and strenghts, otherwise good drugs may be discarded.