Bulbar muscles, those of the face and throat, are affected in spinal muscular atrophy (SMA), and researchers in Germany found that young children with SMA cannot open their mouths as well as age-matched infants and toddlers without this disease. “There is a growing interest in the swallowing and the…
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Limited social interaction and difficulties in accessing needed medications and physical therapy were consequences of the COVID-19 pandemic, parents and grandparents of children with spinal muscular atrophy (SMA) in Sweden reported. In online surveys and chats, family members spoke of having to take charge of their child’s physiotherapy and…
With deep sadness, we announce the passing of our dear friend and longtime contributor, DeAnn Runge. DeAnn had many roles at Bionews, the parent company of SMA News Today. She was a forum moderator, spinal muscular atrophy (SMA) community advocate, and member of our recently established Patient…
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 Phase 3 data as the foundation of our submission, we look forward…
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 — which works by increasing the levels of dopamine — could help ease such…
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 shrinking gray matter was tied to weaker muscles and worse motor function. More research is needed…
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 all ages — a regimen the therapy’s developer says may provide more effective treatment…
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 new approaches for future guidelines in the management of pregnancy and SMA,” the…
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 type 2 disease. That’s according to a real-world study in the…
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, overall, strongly associated with disease severity across two years of follow-up, and…
