A new and noninvasive type of antisense oligonucleotide (ASO) therapy effectively treated spinal muscular atrophy (SMA) in a mouse model with established symptoms, a study reported. Since symptomatic and late-onset SMA patients are the most difficult to treat, “our [therapy] should be considered a great improvement from a clinical…
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Combined treatment with Spinraza and Zolgensma does not markedly improve motor function or breathing ability in children with spinal muscular atrophy (SMA) type 1, a small, single-site study reported. Its scientists highlighted that, regardless of the type of treatment given, earlier treatment leads to better outcomes. The study, “…
Surgery to correct kyphoscoliosis — a combination of two types of abnormal curvature of the spine — allows the successful delivery of Spinraza (nusinersen) into the spinal canal of children with spinal muscular atrophy (SMA) type 1, a case series study shows. However, post-surgery complications occurred in all four described…
Playing wheelchair hockey is associated with better psychological well-being, self-perception of physical abilities, and quality of life in men with spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), a small study in Italy shows. These findings add to some previous studies reporting the health benefits of adaptive sports…
The team of researchers who discovered and developed Evrysdi (risdiplam) — the first at-home oral therapy for spinal muscular atrophy (SMA) — was selected as the winner of two prestigious treatment discovery awards. These are the Society for Medicines Research (SMR)’s 2021 Award for Drug Discovery and the British Pharmacological…
Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and young adults with types 2 and 3 SMA who are unable to walk. Apitegromab will be given as an add-on therapy to…
Spinraza (nusinersen) may restore the normal development of motor neurons — the nerve cells responsible for making muscle cells work — in children with spinal muscular atrophy (SMA), particularly those who start treatment early, a study found. Besides getting motor neurons back onto their developmental trajectory, Spinraza also may slow their…
A song written and performed by an artist with spinal muscular atrophy (SMA) is the latest project to come out of Genentech’s SMA My Way program, a collaboration that allows those in the SMA community to share knowledge and experiences with others affected by the disease, as well…
A CA$414,000 grant was awarded to the Maritimes by Muscular Dystrophy Canada (MDC) and Novartis Pharmaceuticals Canada to further support the implementation of newborn screening for spinal muscular atrophy (SMA). The funding (about $322,600), received by the Maritime Newborn Screening Program (MNSP), will go toward work to…
One of NBC’s newest TV shows, “Ordinary Joe,” is making a mark in the spinal muscular atrophy (SMA) community for including a character with the disease. The drama series follows protagonist Joe Kimbreau, played by James Wolk, through three separate realities stemming from a single decision he made…
