Treatment before birth with RG7916, a compound similar to Evrysdi (risdiplam), improved weight, muscle health, motor function, and survival in a mouse model of severe spinal muscular atrophy (SMA), a study found. However, treatment given only before birth, or prenatally, did not fully preserve numbers of motor neurons…
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The Muscular Dystrophy Association (MDA) brought its Engage Community Seminar to Hershey, Pennsylvania, on July 18, uniting individuals with neuromuscular diseases, caregivers, and medical experts for a day of education and connection. Hosted in collaboration with the Penn State Health Milton S. Hershey Medical Center, the one-day event featured a…
A woman with spinal muscular atrophy (SMA) type 3 experienced a mild decline in motor function after pausing Spinraza (nusinersen) during her pregnancy. After she restarted treatment following delivery, however, her motor function stabilized and her walking endurance partially recovered. Overall, the case suggests that women with milder SMA and better-preserved…
An official decision on whether to approve apitegromab for spinal muscular atrophy (SMA) in the European Union may hinge on upcoming inspection results from U.S. regulators, biopharmaceutical company Scholar Rock has announced. The European Medicines Agency (EMA) is currently reviewing the experimental muscle-strengthening therapy. However, European authorities are…
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…
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…
