Trial of AJ201 in SBMA fully enrolled, results expected this year
A Phase 1b/2a clinical trial testing the experimental therapy AJ201 in people with spinal and bulbar muscular atrophy (SBMA)…
Articles by Marisa Wexler, MS
Zolgensma may safely benefit older, heavier children with SMA: Study
The gene therapy Zolgensma (onasemnogene abeparvovec) did not cause any serious long-term safety problems when used in children with…
Spinraza benefits most type 1 SMA kids on ventilation in Korean study
Treatment with Spinraza (nusinersen) led to modest improvements in motor function for most children with type 1 spinal…
Access needed to gene therapy for SMA outside rich countries: Paper
More work is needed to expand access to the gene therapy Zolgensma (onasemnogene abeparvovec) for people with spinal…
Executive function deficits found in adult SMA type 3 patients: Study
Adults with spinal muscular atrophy (SMA) type 3 showed deficits in certain areas of cognition, particularly executive function or…
2 type 1 SMA babies show signs of restored motor neuron connections
Treatment with Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec) led to increases in the electrical activity of motor neurons…
Breathing problems during sleep often mistaken for apnea in SMA
People with spinal muscular atrophy (SMA) may experience bouts of breathlessness during sleep that can look at first glance…
Measuring arms’ nerve activity may help predict response to Evrysdi
Measuring the electrical activity of a pair of nerves that control arm movement, called the median nerves, could help assess…
Novel splicing mutation found to cause SMA for baby in Cyprus
A never-before-documented mutation in the SMN1 gene was identified as the cause of spinal muscular atrophy (SMA) type 1 for…
Targeted gene mutations test may help ID cause of rare SMA types
A targeted test looking for mutations in known disease-associated genes in spinal muscular atrophy (SMA) was found to be…