SMA types

SMA progression in types 2 and 3 varies without treatment: Study

In the absence of treatment, the long-term progression of spinal muscular atrophy (SMA) types 2 and 3 is highly variable and seems to be influenced by factors including a patient’s age and functional status, according…

Long-term Spinraza found safe, effective across SMA spectrum

Long-term treatment with Spinraza (nusinersen) was associated with improved or stable motor function in patients across the spinal muscular atrophy (SMA) disease spectrum, according to an analysis of real-world registry data. “To date, this is the largest prospective study over the longest observational period of [Spinraza] therapy…

Grip strength may be better test of subtle upper limb changes in SMA

Measures of grip strength or mobility may help to capture subtle changes in upper limb function for children with spinal muscular atrophy (SMA), particularly for patients who are overall less severely affected, according to findings from a recent study. The Revised Upper Limb Module (RULM), which is a standard…