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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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