Author Archives: Marisa Wexler, MS

Scholar Rock has resubmitted its application seeking approval in the U.S. of  its muscle-strengthening agent apitegromab — rejected last year due to manufacturing issues — for the treatment of children and adults with spinal…

The U.S. Food and Drug Administration (FDA) has approved a high-dose regimen of Spinraza (nusinersen) — one that allows patients to receive consistently higher doses of the therapy after a shorter initial treatment loading phase — for…

A combination of approved treatments, alongside early physical therapy, was successfully deployed to help treat a boy with spinal muscular atrophy (SMA) type 0 in Japan. Type 0 is the most severe…

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,…