Different types of nerve cells respond differently when activity of a gene linked to spinal muscular atrophy (SMA) is reduced — and responses can vary even among nerve cells of the same type, according to a new study using nematode worms. These findings shed new light on the underlying biology…
News
August is SMA Awareness Month, and advocates around the world are gearing up to raise awareness about spinal muscular atrophy (SMA) by sharing stories, raising funds, and connecting as a community. SMA News Today will mark the month with a series of videos in which people with SMA and…
A community-driven campaign called SMAshing My Limits And Going Places aims to make it easier for people with spinal muscular atrophy (SMA) to find accessible places to stay when they travel. The campaign is spearheaded by Becoming Rentable, an online platform dedicated to helping people with disabilities find…
A combined newborn screening test for two rare diseases — spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID), a condition that leaves infants with little or no working immune systems — correctly identified every affected baby, with no missed or false diagnoses. The five-year Italian pilot screened 32,289…
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…
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…
