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A “significant” economic burden is associated with spinal muscular atrophy (SMA), although differences in healthcare costs and resource use are evident among patients by SMA type, a large review study found. Not surprisingly, the greater burden is among patients with SMA type 1, one of this disease’s most…
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…
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, “…
Throughout Michael’s physical therapy treatments, there have been several progressions of seated balance activities that we have worked on as he has demonstrated improved abilities to perform these activities. When we were first able to transfer Michael to the therapy table, all that was performed in a seated position was…
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…
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