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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…
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,…
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.
A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2Â gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived from a spinal muscle…
Treatment with OAV101 IT, a formulation of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s delivered directly into the spinal canal, safely led to motor improvements for children with spinal muscular atrophy (SMA) type 2 who…
Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read stories we published last year, each with a brief…
One of my patients, Michael, who has spinal muscular atrophy, has continued to experience increased pain and pressure along his coccyx and into his right hip while sitting in his wheelchair all day. He is unable to move his lower extremities independently and is reliant on sitting in…
Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has “radically changed the…
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height may help predict the risk of scoliosis in people with spinal muscular atrophy (SMA) who have received…
SMA Europe is launching an initiative to share personal stories of how treatment affects the lives of people with spinal muscular atrophy (SMA). The initiative comes as part of OdySMA, an SMA…
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