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…
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…
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