Recently, in the SMA News Today Forums, a handful of members shared that they were embarking on new treatment journeys with both Spinraza (nusinersen) and Evrysdi (risdiplam). Reading such posts makes me feel a level of happiness I don’t often feel for strangers on…
It’s now been more than four years since Spinraza (nusinersen) was first approved by the U.S. Food and Drug Administration, and presymptomatic children with spinal muscular atrophy (SMA) who received the therapy as part of the Phase 2 NURTURE trial are growing up fast and, by all accounts, showing…
Patient advocacy groups, a university, and pharmaceutical companies are among the 12 new partners of the European Alliance for Newborn Screening in Spinal Muscular Atrophy, also known as the SMA NBS Alliance. SMA Europe founded the alliance last year in an effort to include SMA in all newborn…
Nearly a month into 2021, I think it’s safe to say we’re all glad to have left 2020 behind us. It certainly wasn’t an easy year. Its hardships likely will go down in the history books. Around the globe, we saw a devastating pandemic, raging wildfires, and a…
Improvement in motor function, breathing, and ease of administration are among the factors that people with spinal muscular atrophy (SMA) and their caregivers value most when deciding on available treatments, a new study reports. The study, “Treatment preference among patients with spinal muscular atrophy (SMA): a…
Among children with neuromuscular disorders, those with spinal muscular atrophy type 1 (SMA 1) appear to be more vulnerable to more severe COVID-19 symptoms, a small study suggests. However, the course of disease in children with neuromuscular disorders overall may not be as…
Four measures of motor ability — including one of continuous upper limb activity that can be done at the home — reliably detect significant functional changes over one or two years in people with spinal muscular atrophy (SMA) types 2 and 3, according to a natural history…
When I say, “I don’t need my SMA to be fixed,” I mean it from the bottom of my heart. Last month, I set sail with my first dose of the new disease-modifying therapy Evrysdi (risdiplam). It was a choice that required careful consideration of my options and…
A free resource booklet, published by Cure SMA, may help people with spinal muscular atrophy (SMA) to better understand the possible risks and benefits of combining different treatments for the disease. The booklet, titled “Scientific Considerations for Drug Combinations,” is available online. The past several years…
SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal muscular atrophy (SMA) throughout 2020. We look forward to reporting more news to patients, family members, and caregivers dealing with SMA during 2021. Here are the top 10 most-read articles…