Healthcare professionals who specialize in managing spinal muscular atrophy (SMA) are prone to putting off tasks related to patient care, likely because they are stressed and exhausted, a study found. A research team in Spain…
Using wearable sensors during clinical tests may help doctors objectively measure changes in movement ability for people with spinal bulbar muscular atrophy (SBMA), a study reports. “These findings support incorporating wearable sensors into clinic assessments and encourage…
Problems with swallowing, also called dysphagia, remain a concern for many children with spinal muscular atrophy (SMA) type 1 despite available treatments, a real-world study reports. Researchers examined swallowing in patients with SMA…
The use of disease-modifying therapies (DMTs) — all widely approved in the last 10 years — may help children with spinal muscular atrophy (SMA) maintain nutrition and prevent stunted growth, an Australian study suggests.
What is Spinraza for SMA? Spinraza (nusinersen) is an injection therapy widely approved for treating adults and children with spinal muscular atrophy (SMA). It was the first therapy approved to target the underlying causes of SMA. Mutations in the SMN1 gene, which contains instructions for cells to produce…
The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results…
Up to three years of treatment with Evrysdi (risdiplam) appears safe and was associated with presymptomatic children with spinal muscular atrophy (SMA) reaching and maintaining key developmental milestones, according to Phase 2 study…
A high-dose regimen of Spinraza (nusinersen) that is up for regulatory approval in the U.S. may help stabilize or improve motor function for people with spinal muscular atrophy (SMA), pooled results from two clinical trials…
Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in children…
Genetic screening for spinal muscular atrophy (SMA) in newborns may allow treatment to begin earlier, which could help support motor development, a study suggests. This “adds to the evidence that [newborn screening] for SMA is…
Get regular updates to your inbox.