Author Archives: Marisa Wexler, MS

The one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) is now approved in the European Union for people with spinal muscular atrophy (SMA) ages 2 and older — a regulatory decision that will make…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to salanersen, a new, more potent Biogen treatment for spinal muscular atrophy (SMA) that’s now in late-stage testing. The treatment candidate, being evaluated…

Spinraza (nusinersen) is able to help slow lung function decline in people with spinal muscular atrophy (SMA), even if treatment is started when patients are already experiencing disease symptoms, a new study shows.

The U.S. Food and Drug Administration (FDA) is expected to decide in the coming months whether or not to approve apitegromab, an experimental muscle-strengthening agent designed to improve motor function in people with…

A new high-dose regimen of Spinraza (nusinersen) was recently approved in the U.S. as a treatment for spinal muscular atrophy (SMA), with clinical trial data showing a safety profile broadly consistent with the original 12…

A European Medicines Agency (EMA) panel recommended that the one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) be approved for patients with spinal muscular atrophy (SMA) ages 2 and older. The Committee for Medicinal Products for Human Use (CHMP)…

Surgery to implant magnetically controlled growing rods (MCGRs) is a safe and effective option for children with spinal muscular atrophy (SMA) type 1 and scoliosis, according to a study. “MCGR represents a viable and…

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…