Adults with spinal muscular atrophy (SMA) who start treatment with Spinraza (nusinersen) tend to see the most dramatic…
Articles by Marisa Wexler, MS
Children with spinal muscular atrophy (SMA) who are treated with Zolgensma (onasemnogene abeparvovec-xioi) tend to have more dramatic…
A second-generation gene therapy, administered directly into the brain, led to faster and more pronounced improvements on motor function in…
The side effects that some patients experience on Evrysdi (risdiplam), an approved treatment for spinal muscular atrophy (SMA),…
Treatment with Evrysdi (risdiplam) in spinal muscular atrophy (SMA) patients who previously received other therapies was found to…
In adults with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen) in a clinical trial was tolerated well…
Twin girls with type 0 spinal muscular atrophy (SMA) — the most severe form of the disease, which usually…
Children and adolescents in New Zealand with spinal muscular atrophy (SMA) will be able to access Evrysdi (risdiplan)…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA)…
A group of proteins called the SNARE complex, which normally helps regulate how nerve cells receive chemical messages from other…