Author Archives: Margarida Maia, PhD

The U.S. Food and Drug Administration (FDA) has declined to approve a higher dose of nusinersen — the medication in Biogen’s long-approved therapy Spinraza — for the treatment of spinal muscular atrophy (SMA). According to Biogen, no…

An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study shows. The study, “The effect of coadministration of D156844 and AR42 (REC-2282) on…

The use of salbutamol may safely improve upper limb strength, as well as provide other benefits, in people with spinal muscular atrophy (SMA), particularly among young children, according to data from a new meta-analysis — though…

A quantitative MRI scan, which shows detailed images of muscles, can be used to track changes seen with Spinraza (nusinersen) treatment in children with spinal muscular atrophy (SMA), according to a new study from China.

Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized, ride-on cars may boost their confidence, help them connect with others, and make them feel more included, a…

After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed improved motor function and more independence in tasks involving their arms and hands, a study from China has found.

Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type 3 and to be safe in non-human primates, supporting its move into clinical testing. Genecradle Therapeutics is…

It can be difficult for genetic counselors in the U.S. and Canada to explain indeterminate results of prenatal screening for spinal muscular atrophy (SMA), as patients are often confused or distressed before appointments, and misunderstandings are common among…

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…

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…