FDA approves new tablet version of oral SMA treatment Evrysdi
The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral treatment…
Articles by Marisa Wexler, MS
Cure SMA seeks $10M in DOD funding in advocacy campaign
Cure SMA is leading an advocacy campaign for new research funding from the U.S. Department of Defense (DOD) that…
Scholar Rock asks FDA to approve apitegromab for SMA
Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy…
Model predicts scoliosis risk in patients receiving SMA treatment
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height…
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…
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…