The U.S. Food and Drug Administration (FDA) has once again found problems at a manufacturing facility that was included on Scholar Rock‘s application seeking approval of apitegromab, its experimental muscle-strengthening agent for spinal muscular atrophy (SMA). In light of the continued issues at the facility, Scholar Rock…
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Children with spinal muscular atrophy (SMA) have a higher risk than their peers in the general population of developing severe respiratory syncytial virus (RSV) infections, a nationwide study from Taiwan found. SMA kids were more likely to be hospitalized, require admission to an intensive care unit (ICU), and use…
In a few weeks, SMA News Today will host a free webinar exploring aspects of life with spinal muscular atrophy (SMA) that are rarely discussed in the doctor’s office, including romantic relationships, careers, and travel.
Registry studies can provide a valuable glimpse into how outcomes are evolving in rare diseases such as spinal muscular atrophy (SMA), but a lack of complete data can limit their usefulness, a study found. The researchers called for “improved processes and governance for communication and collaboration across registries [and]…
In infants diagnosed via newborn screening with spinal muscular atrophy (SMA), measures of nerve function may be a useful way to quickly evaluate disease activity and track how these young patients respond to SMA treatments. That’s according to a new U.S. study, which found that results of tests of…
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…
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…
