Author Archives: Marisa Wexler, MS

Mutations that cause spinal muscular atrophy (SMA) disrupt the development of spinal cord cells, leading to fewer nerve cells in early development, according to a new study in cell models. The findings highlight potential…

Biogen has launched a trio of trials to test its new drug candidate for spinal muscular atrophy (SMA), salanersen, which aims to be a more convenient successor to the company’s approved SMA therapy…

The first patient has been dosed in a clinical trial testing GB221, a next-generation gene therapy for spinal muscular atrophy (SMA) type 1. “I would like to express our gratitude for the young child and family…

Spinal muscular atrophy (SMA) is associated with multi-organ changes in a protective protein modification that helps guard cells against molecular damage, according to a study in mice. The researchers also found that treatment with antisense oligonucleotides —…

Treatment with Evrysdi (risdiplam) may modestly improve arm function for adults with spinal muscular atrophy (SMA), a new study from the Czech Republic and Slovakia indicates. Findings suggest that adults with SMA generally…

Children with spinal muscular atrophy (SMA) often struggle to get a good night’s sleep — and their caregivers also commonly report feeling sleep-deprived, according to a new study. “Sleep problems have potentially critical consequences for…

Children with spinal muscular atrophy (SMA) who start on a disease-modifying therapy (DMT) — four have been approved in the last decade — before developing symptoms are usually able to feed by mouth,…

Treatment with a high-dose regimen of Spinraza (nusinersen) was shown to safely improve motor function in children with spinal muscular atrophy (SMA) in the Phase 2/3 DEVOTE clinical trial. Most patients who switched from the…

A rapid test using zebrafish was able to show that two children identified by genetic testing as possibly having spinal muscular atrophy (SMA) did not actually have the progressive disease, according to a new study.

Treatment with gene therapy significantly prolonged survival and improved motor function in a mouse model of spinal muscular atrophy with respiratory distress type 1 (SMARD1), researchers report. The gene therapy tested in this lab…