Marisa Wexler, MS,  senior science writer—

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,…

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,…

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…

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…