While effective treatment is lengthening life and improving its quality, children with spinal muscular atrophy (SMA) type 1 often have abnormal spinal curvatures like scoliosis that require long-term care, a study highlights. “Novel therapies have made this [SMA] a treatable condition, resulting in increased life expectancy,” the researchers, all…
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The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral treatment for spinal muscular atrophy (SMA). “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date,” Levi Garraway, MD,…
Treatment with Spinraza (nusinersen), which was the first disease-modifying therapy to be approved for spinal muscular atrophy (SMA) back in 2016, does not lead to significant kidney dysfunction in people with SMA types 1 and 2, according to a new study from Turkey. Further, the researchers did not…
Metabolic abnormalities, including the buildup of fat in the liver and other issues, occur before the loss of motor neurons, according to a study in a mouse model of spinal muscular atrophy (SMA). The mice, with reduced levels of the SMN protein, exhibited decreased levels of glucose (sugar) in…
Cure SMA is leading an advocacy campaign for new research funding from the U.S. Department of Defense (DOD) that would be dedicated to addressing unmet challenges for people with spinal muscular atrophy (SMA). The campaign is seeking $10 million in SMA-dedicated funding from the DOD’s medical research program,…
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…
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…
