News

Liver damage may occur as a side effect of the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), but it usually resolves within a few months of treatment, a study showed. The researchers also found that simultaneous treatment with Zolgensma and Spinraza (nusinersen) does not seem to…

Starting Evrysdi (risdiplam) very early — just 12 days after his birth and before his symptoms appeared — was safe and helped a boy with spinal muscular atrophy (SMA) achieve key motor milestones, such as sitting and walking, within the normal time window. Adding to clinical evidence that…

Treatment with Spinraza (nusinersen) or Evrysdi (risdiplam) may help preserve upper limb motor function in children and young adults with spinal muscular atrophy (SMA) type 2, a natural history study in Spain shows. In the study, the researchers saw that upper limb motor function, measured using a clinical…

Not all healthy children acquire full feeding skills by 2 years, according to a study that applied an instrument called DySMA — which assesses swallowing in infants and children with spinal muscular atrophy (SMA) who are undergoing treatment — to healthy children in a study to enable more…

Motor improvements in adults with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) paralleled a boost in the strength and duration of electrical impulses along nerves that supply the muscles, a study shows. In an SMA mouse model, Spinraza restored dormant motor neurons, the nerve cells that aid…

Testing patient DNA with advanced methods like HapSMA — which can “[analyze] … SMN and its surrounding genes” — can reveal otherwise undetectable genetic changes, such as gene conversion, that affect how severe spinal muscular atrophy (SMA) is, according to a study by researchers in the Netherlands. These novel…

Treatment with Spinraza (nusinersen) and physical therapy that focused on posture improved motor skills in a young boy with spinal muscular atrophy (SMA) type 0 and prevented the progression of scoliosis. That’s according to a case report in Japan that demonstrated the boy was able to sit…

Three men with late-onset spinal muscular atrophy (SMA) undergoing treatment with the approved therapy Evrysdi (risdiplam) were able to conceive with their partners, a case series reported. None of the pregnancies were planned, and the researchers noted there were no missed treatment doses. Among the three, one pregnancy…

A year of disease-modifying therapies (DMTs) improved motor function, particularly fine motor skills, in people with spinal muscular atrophy (SMA) who could sit, but not walk due to disease progression before they had access to treatment, a study reports. These functional improvements were independent of treatment-related gains in…

Powered, ride-on toy cars to help children with spinal muscular atrophy (SMA) type 1 be more mobile must be adapted for easy access and match a child’s motor abilities, according to parents who took part in a 12-week training program in Spain. The parents also said it’s important to…