SMA webinar to explore relationships, careers, and travel beyond the clinic
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…
Author Archives: Marisa Horak, MS
Incomplete registry data limits value of SMA studies
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…
Nerve function tests may help track response to SMA treatment in babies
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…
SMA-related gene activity affects nerve cells differently in worm study
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…
Global events planned to highlight SMA Awareness Month
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…
Campaign expands accessible rental options for SMA vacationers
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…
European review of SMA therapy awaits upcoming results from US facility check
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 expands newborn screening to detect SMA before symptoms appear
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…
Study links metabolic flaws in spinal muscles to scoliosis in SMA
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…
Pioneering SMA champion to be honored for her disability advocacy
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) —…
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- SMA webinar to explore relationships, careers, and travel beyond the clinic
- Incomplete registry data limits value of SMA studies
- Community Bingo Challenge
- Nerve function tests may help track response to SMA treatment in babies