Spinal muscular atrophy (SMA) is a rare genetic condition characterized by the progressive loss of motor neurons, which are the specialized nerve cells that control voluntary movement, leading to muscle weakness and atrophy, among other symptoms. There are several types of SMA, based on the affected gene, age…
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Novartis Pharmaceuticals‘ investigational compound BVS857 significantly improved muscle volume in patients with X-linked type spinal muscular atrophy (SMA) over a short period of time, Phase 2 clinical results show. However, the compound failed to improve muscle strength and function. These findings were reported in the study, “…
Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy (SBMA) develops before symptoms appear, a Japanese study reports. The research on the waste product, creatinine, appeared in the journal Neurology. The title of the…
Levels of a male hormone produced by the adrenal glands could provide useful information on the severity of spinal and bulbar muscular atrophy, or SBMA, a study reports. The identified the hormone as DHEA sulfate. Researchers’ study, published in the Journal of Neurology, is titled “The metabolic…
The patent application for gene therapy candidate INT41, developed by Vybion for the treatment of Huntington’s disease, spinal muscular atrophy (SMA), and other neurodegenerative diseases, was granted Track One status from the U.S. Patent and Trademark Office (USPTO). The patent application covers the treatment’s composition and methods of use.
Patients with spinal and bulbar muscular atrophy (SBMA) may be at risk of developing respiratory complications due to a decline in the levels of genes responsible for muscle strength, according to a new study. The authors assert that monitoring the breathing decline in these patients may help provide better clinical…
Children with spinal muscular atrophy (SMA) who start on a disease-modifying therapy (DMT) — four have been approved in the last decade — before developing symptoms are usually able to feed by mouth, but SMA patients who already have swallowing difficulties before beginning treatment often continue to have…
Treatment with a high-dose regimen of Spinraza (nusinersen) was shown to safely improve motor function in children with spinal muscular atrophy (SMA) in the Phase 2/3 DEVOTE clinical trial. Most patients who switched from the originally approved dose to the high-dose regimen experienced improvements in motor function scores,…
First-line Zolgensma (onasemnogene abeparvovec-xioi) outperformed first-line Spinraza (nusinersen) in achieving a more sustained clinical response and reducing the need for supportive care among young children with spinal muscular atrophy (SMA) type 1, according to a head-to-head analysis. Still, both therapies led to comparable, clinically meaningful improvements in…
Nearly all children with spinal muscular atrophy (SMA) who were started early on Evrysdi (risdiplam) in a clinical trial — as newborns, before the onset of symptoms — are now walking after three years on the approved oral therapy. Further, most of the youngsters also have no need…