In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma…
Articles by Marisa Wexler, MS
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) didn’t cause any unexpected safety issues among children with spinal muscular…
Children with spinal muscular atrophy (SMA), even those with substantial physical disability, tend to report that their quality of…
A Phase 1b/2a clinical trial testing the experimental therapy AJ201 in people with spinal and bulbar muscular atrophy (SBMA)…
The gene therapy Zolgensma (onasemnogene abeparvovec) did not cause any serious long-term safety problems when used in children with…
Treatment with Spinraza (nusinersen) led to modest improvements in motor function for most children with type 1 spinal…
More work is needed to expand access to the gene therapy Zolgensma (onasemnogene abeparvovec) for people with spinal…
Adults with spinal muscular atrophy (SMA) type 3 showed deficits in certain areas of cognition, particularly executive function or…
Treatment with Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec) led to increases in the electrical activity of motor neurons…
People with spinal muscular atrophy (SMA) may experience bouts of breathlessness during sleep that can look at first glance…