News

Biogen is very pleased with the “striking” results seen thus far in clinical testing with its therapy candidate salanersen — designed to have high efficacy in people with spinal muscular atrophy (SMA) — and is engaging with health authorities on the design of further trials that might provide…

August is Spinal Muscular Atrophy (SMA) Awareness Month where community members will join together on behalf of patient advocacy and research funding to help find a cure for the rare genetic condition. “August is our time to shine a brighter spotlight on the needs, wants, hopes, and experiences of…

Most parents and potential parents in the U.K. support screening newborns for spinal muscular atrophy (SMA) as part of the National Health Service (NHS) Newborn Screening Programme, according to a survey developed and funded by Novartis. “We believe that this government has an opportunity to make newborn screening…

A New York-based scientist has been awarded for his pioneering work that led to the development of Spinraza (nusinersen), the first RNA-based therapy to target the underlying cause of spinal muscular atrophy (SMA). Adrian R. Krainer, PhD, a professor at Cold Spring Harbor Laboratory, in New York, will formally…

A higher-dose regimen of nusinersen, currently up for approval in the U.S., may provide further benefits to people with spinal muscular atrophy (SMA) relative to the approved Spinraza regimen, according to its developer, Biogen. Nusinersen is marketed as Spinraza. The Phase 3 DEVOTE clinical trial (NCT04089566) part C showed…

A novel mutation in the ASAH1 gene was identified as the cause of spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME) in a 4-year-old boy, according to a recent report. Despite the genetically confirmed diagnosis, the boy showed no signs of myoclonic seizures, or brief muscle spasms, which…

Molecular markers in the liquid that surrounds the brain and spinal cord, called the cerebrospinal fluid (CSF), may help predict how well people with type 2 or 3 spinal muscular atrophy (SMA) respond to Spinraza (nusinersen), a study reports. The study’s researchers found that certain CSF molecules involved…

A group of questionnaires called PROfuture can meaningfully measure a variety of symptoms of spinal muscular atrophy (SMA) that impact patients, a study reported. “The PROfuture questionnaires, developed by patients for patients in the current era of new treatments for SMA, can help to better characterize the impact of…

Treatment with Evrysdi (risdiplam) is generally safe and effective at improving or stabilizing motor and lung function in adults with spinal muscular atrophy (SMA) for up to two years, according to a real-world study in Belgium. Results also suggested easing of difficulty swallowing and fatigue, as well as…

Interim results from a Phase 1 clinical trial show salanersen, Biogen’s treatment candidate for spinal muscular atrophy (SMA), slowed neurodegeneration, and improved motor function, in children with the genetic disease. The data also demonstrated that four of eight SMA children treated for at least one year attained new…