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…
The activity of many genes may be dysregulated in spinal muscular atrophy (SMA), but the way the disease affects genes seems to vary across different types of tissues in the body, a study found. “Our comparative meta-analysis identified only few genes and pathways that were consistently dysregulated in SMA…
Taldefgrobep alfa did not significantly improve motor function across the total population of treated spinal muscular atrophy (SMA) patients in a pivotal Phase 3 clinical trial, but significant improvements were seen among Caucasian participants. That’s according to trial results announced by the therapy’s developer, Biohaven Pharmaceuticals. The…
Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with spinal muscular atrophy (SMA), a study reported. The study, “Performance fatigability in adults with spinal muscular atrophy treated long-term with nusinersen,” was published in Muscle and Nerve. SMA is…
Twin girls with spinal muscular atrophy (SMA) type 0 are still alive with normal motor function more than two years after treatment with Zolgensma (onasemnogene abeparvovec-xioi). This finding dramatically differs from the typical progression of SMA type 0, the rarest form of the disease, where symptoms…
Researchers have developed a new spinal muscular atrophy (SMA) screening method that’s quick, inexpensive, and easy to use with standard lab equipment. The team described their method in a paper, “A rapid and easy-to-use spinal muscular atrophy screening tool based on primers with high specificity and amplification…
Should results of a Phase 3 trial of apitegromab, a muscle-directed therapy for spinal muscular atrophy, be positive when released later this year as expected — supporting the add-on therapy being approved — apitegromab could be available to U.S. patients in 2025, Scholar Rock, its developer, announced.
Standardized measures to track the severity of spinal muscular atrophy (SMA) don’t adequately capture aspects of the disease that have major impacts on patients and their families, such as fatigue and the need for medical appointments that can be challenging to schedule, a study reports. “This study provides very…
In people with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), levels of pro-inflammatory signaling molecules tend to decrease while levels of anti-inflammatory signaling molecules tend to increase in the months after starting treatment, a study has found. Findings also suggested that changes in certain inflammation-regulating molecules may…
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