News

COVID-19 made life with SMA more difficult, parents and doctors say

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…

In memoriam, DeAnn Runge: Sept. 4, 1979-Jan. 21, 2025

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 asks FDA to approve apitegromab for SMA

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…

High adherence to Evrysdi linked to lower patient healthcare costs

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 creatinine levels may best mark progression, therapy response

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…

Fertility issues common in men with SMA, study finds

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. These conditions were particularly common in SMA type 2 patients. “To…