Isis Pharmaceuticals reports that treatment with its antisense drug, which changes how SMN2 genes are "read" by cells, results in functional improvement
posted on May 1, 2014 - 3:53pm
Update (Aug. 1, 2014): The phase 2 infant study of ISIS-SMNRx is now closed to new participants. However, a phase 3 study of this drug has opened. See ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA.