SMA Europe inviting patients to talk of how treatment has changed life
SMA Europe is launching an initiative to share personal stories of how treatment affects the lives of people with…
Articles by Marisa Wexler, MS
SMA’s effects on gene activity vary across tissues: Study
The activity of many genes may be dysregulated in spinal muscular atrophy (SMA), but the way the disease affects…
Taldefgrobep alfa aids muscle mass for Caucasian patients in pivotal trial
Taldefgrobep alfa did not significantly improve motor function across the total population of treated spinal muscular atrophy (SMA) patients…
SMA treatment Spinraza eases fatigability in patients who can walk
Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with spinal muscular…
Zolgensma leads to normal motor function in twins with SMA type 0
Twin girls with spinal muscular atrophy (SMA) type 0 are still alive with normal motor function more than two…
Researchers develop cheap, simple SMA screening test
Researchers have developed a new spinal muscular atrophy (SMA) screening method that’s quick, inexpensive, and easy to use with…
Muscle gains with apitegromab still seen in TOPAZ, Phase 3 data on way
Should results of a Phase 3 trial of apitegromab, a muscle-directed therapy for spinal muscular atrophy, be positive…
Mobility measures miss key ways SMA impacts patients: Study
Standardized measures to track the severity of spinal muscular atrophy (SMA) don’t adequately capture aspects of the disease that…
Inflammatory signaling molecules may predict response to Spinraza
In people with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), levels of pro-inflammatory signaling molecules tend to…
Spinraza aids motor function for SMA children at center in China
Treatment with Spinraza (nusinersen) led to improvements in motor function for nearly all children with spinal muscular atrophy…