After five years of daily treatment with Evrysdi (risdiplam), 90% of children with spinal muscular atrophy (SMA) type 1 were alive, and most had reached motor milestones, such as sitting without support, that are rarely seen in untreated children with this severe disease. These are the final published…
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Daily treatment with Evrysdi (risdiplam) may help stabilize or improve motor function and improve well-being in adults with severe forms of spinal muscular atrophy (SMA), according to a real-world study from the Netherlands. The study showed Evrysdi may benefit people with longstanding SMA …
Muscle ultrasound helps assess muscle health and its association with motor function in children with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen), according to a recent study. While the findings varied by muscle group and SMA type, ultrasound data generally indicated that children with SMA exhibited muscle wasting…
The one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) is now approved in the European Union for people with spinal muscular atrophy (SMA) ages 2 and older — a regulatory decision that will make the injection therapy available to SMA patients of all ages. According to therapy developer Novartis,…
A study in adults with spinal muscular atrophy (SMA) found that Evrysdi (risdiplam) helps the body produce more functional SMN protein, and also revealed molecular differences between SMA types 2 and 3 that may affect how the disease progresses and responds to treatment. The findings, which showed differences…
Weak bones in people with spinal muscular atrophy (SMA) may be driven by disrupted muscle-to-bone communication, a study in China showed. The researchers suggested that low levels of SMN protein — a hallmark of SMA — drive low levels of another protein, SNAP23, which in turn impairs the release…
SMA Europe is accepting proposals for research projects that can help scientists better understand spinal muscular atrophy (SMA) and improve outcomes for people living with the condition. This is the organization’s 13th biennial call for proposals. For this round of funding, projects about regeneration of the motor…
Omaveloxolone, a therapy designed to promote anti-stress responses in cells, may help correct stress-related dysfunction in certain cells from people with spinal muscular atrophy (SMA), according to a lab-based study. The treatment works by boosting levels of proteins that help fight stress, including several in a signaling pathway called…
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) helped children with spinal muscular atrophy (SMA) gain or maintain motor milestones and motor function, a real-world study in Japan found. The RESTORE patient registry (NCT04174157) is assessing the long-term safety and efficacy of Zolgensma in clinical settings across 99 locations in the…
The migraine medication flunarizine may help protect nerve cells in spinal muscular atrophy (SMA) by correcting abnormal levels of microRNA, small molecules that help regulate gene activity. Researchers identified widespread microRNA abnormalities in cells and in the spinal cords of SMA mice, particularly at the earliest stages of disease.
