Trial of implantable device to help deliver Spinraza soon to start
The U.S. Food and Drug Administration (FDA) has given Alcyone Therapeutics the go-ahead to start a clinical trial testing ThecaFlex…
Articles by Marisa Wexler, MS
Spinraza boosts upper arm, shoulder function in adults with SMA: Study
Adults with spinal muscular atrophy (SMA) who start treatment with Spinraza (nusinersen) tend to see the most dramatic…
Motor benefits of Zolgensma greater when given early: Study
Children with spinal muscular atrophy (SMA) who are treated with Zolgensma (onasemnogene abeparvovec-xioi) tend to have more dramatic…
2nd-generation gene therapy led to better outcomes in early study
A second-generation gene therapy, administered directly into the brain, led to faster and more pronounced improvements on motor function in…
High-dose Evrysdi’s side effects may be due to how it affects genes
The side effects that some patients experience on Evrysdi (risdiplam), an approved treatment for spinal muscular atrophy (SMA),…
AAN 2023: Evrysdi effective in already treated SMA patients in trial
Treatment with Evrysdi (risdiplam) in spinal muscular atrophy (SMA) patients who previously received other therapies was found to…
AAN 2023: Spinraza shows benefits in clinical trial of SMA adults
In adults with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen) in a clinical trial was tolerated well…
AAN 2023: Type 0 SMA twins alive 2 years after Zolgensma treatment
Twin girls with type 0 spinal muscular atrophy (SMA) — the most severe form of the disease, which usually…
Evrysdi wins public funding OK for children with SMA in New Zealand
Children and adolescents in New Zealand with spinal muscular atrophy (SMA) will be able to access Evrysdi (risdiplan)…
Motor gains seen with Zolgensma, especially when given early
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA)…