News

Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy designed to boost motor function in people with spinal muscular atrophy (SMA). “With the strength of our…

Problems in how dopamine and other chemical messengers work in nerve cells can cause issues with posture in spinal muscular atrophy (SMA), according to a new study in mice that also suggests that levodopa therapy —…

Coccyx, or tailbone, mobilization, internal and external, has been a helpful treatment for one of my patients, reducing  tailbone pain. For Michael, who has spinal muscular atrophy, treatments change as his body and other therapies evolve and as his disease progresses. Recently, we scheduled an…

Measuring the amount of gray matter in the spinal cord could help doctors track spinal muscular atrophy (SMA) over time and see how well treatments are working, according to a study that found…

Regulators in the U.S. and the European Union have agreed to review Biogen’s applications seeking approval of a higher dose of Spinraza (nusinersen) for all types of spinal muscular atrophy (SMA) in individuals of…

A 33-year-old woman with spinal muscular atrophy (SMA) type 3 became pregnant while on Spinraza (nusinersen) treatment, and subsequently gave birth to a healthy child, according to a case report. The case “helps to elucidate…

Nonadherence of spinal muscular atrophy (SMA) patients to their prescribed regimen of Evrysdi (risdiplam) — an oral solution generally taken once daily after a meal — is associated with higher healthcare costs, particularly in patients with…

Blood levels of creatinine, a byproduct of normal muscle function, may be a potential biomarker of disease severity and treatment response in adults and adolescents with spinal muscular atrophy (SMA), a study in China shows. Creatinine levels,…

Fertility issues and low sperm counts are common in men with spinal muscular atrophy (SMA), particularly in those with more severe disease, according to a study from France. Almost two-thirds (65.9%) of SMA patients had fertility disorders.

A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived from a spinal muscle…