Surgery to implant magnetically controlled growing rods (MCGRs) is a safe and effective option for children with spinal muscular…
Articles by Marisa Wexler, MS
Scholar Rock has resubmitted its application seeking approval in the U.S. of its muscle-strengthening agent apitegromab — rejected…
The U.S. Food and Drug Administration (FDA) has approved a high-dose regimen of Spinraza (nusinersen) — one that allows…
A combination of approved treatments, alongside early physical therapy, was successfully deployed to help treat a boy with spinal…
Mutations that cause spinal muscular atrophy (SMA) disrupt the development of spinal cord cells, leading to fewer nerve cells…
Biogen has launched a trio of trials to test its new drug candidate for spinal muscular atrophy (SMA),…
The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular…
Spinal muscular atrophy (SMA) is associated with multi-organ changes in a protective protein modification that helps guard cells against…
Treatment with Evrysdi (risdiplam) may modestly improve arm function for adults with spinal muscular atrophy (SMA), a new…
Children with spinal muscular atrophy (SMA) often struggle to get a good night’s sleep — and their caregivers also…