England is rolling out newborn screening for spinal muscular atrophy (SMA), which will allow all babies born in the country to be tested for the rare genetic disease shortly after birth. According to a government press release, the program is already ahead of schedule, and some labs will…
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The spinal muscles of spinal muscular atrophy (SMA) patients who also have scoliosis show abnormalities in molecular activity, particularly in biological processes that cells use to metabolize sugar molecules and protein building blocks, according to a study. The researchers also found that metabolic activity in spinal muscles differs on each…
A woman with spinal muscular atrophy (SMA) who has become a champion for those living with disabilities — as an author, a podcast host, and a vice president of the Muscular Dystrophy Association (MDA) — will be honored for her advocacy work as a recipient of this year’s…
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…
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…
