News

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…

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…

A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived from a spinal muscle atrophy (SMA) patient, researchers report. The addition of the protein, raising SMN protein levels (expression), in…

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 were at least 2 years old,  top-line results from the Phase 3 STEER…

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 description. We’re excited to continue serving the SMA community 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 course of … type I SMA,” with “much higher survival,” according to a nationwide…

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 disease-modifying treatment. “We trained a scoliosis classifier, demonstrating how data give rise…

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 Europe project that tracks access to treatments and care for SMA patients across Europe, with…

Swallowing function may have improved in spinal muscular atrophy (SMA) patients since disease-modifying therapies (DMT) have become available, a review study suggests. The analysis showed problems with swallowing were prevalent in the years before the emergence of DMTs and that studies after the treatments became available indicated stabilizations…