Author Archives: Marisa Wexler, MS

Nearly all children with spinal muscular atrophy (SMA) who were started early on Evrysdi (risdiplam) in a clinical trial — as newborns, before the onset of symptoms — are now walking after three…

The U.S. Food and Drug Administration (FDA) has declined to approve apitegromab as a treatment for spinal muscular atrophy (SMA). The FDA said it could not approve developer Scholar Rock‘s application at this time…

Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the end of the year if the U.S. Food and Drug Administration (FDA) approves it next month…

Treatment with AJ201 was well tolerated and showed signs of improving physical activity in a clinical trial involving people with spinal and bulbar muscular atrophy (SBMA), according to new data announced by developer Annji Pharmaceutical.

Injecting medicines into the amniotic fluid — the liquid that surrounds a developing fetus during pregnancy — may be a viable strategy for treating spinal muscular atrophy (SMA) before birth, according to a new…

A study found that people with spinal muscular atrophy (SMA) have metabolic alterations due to impairments in how their bodies use fatty acids for energy. The results suggested a carbohydrate-rich diet may help normalize metabolism in SMA patients. The…

Liver damage may occur as a side effect of the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), but it usually resolves within a few months of treatment, a study showed. The researchers also found…