News

Higher-dose nusinersen may further improve SMA outcomes: Developer

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…

New ASAH1 mutation found to cause rare SMA type in boy, 4

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…

SMA patient impacts measured by new PROfuture questionnaire set

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 salanersen shows benefits for SMA children in trial

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…

Gains found over 4 years with Spinraza treatment in real world

A real-world study of Spinraza (nusinersen) use in Switzerland among individuals with spinal muscular atrophy (SMA) found that, while patients tended to experience motor function benefits mostly in their first year of treatment, many continued to see gains or disease stabilization for four years. Spinraza treatment had the…

Muscle composition improves for children on Spinraza: Study

Muscle-related body composition measures improved in children with spinal muscular atrophy (SMA) types 2 and 3 who were treated with Spinraza (nusinersen), and the improvements correlated with better motor function, a study found. “Indicators of muscle mass increased in children with SMA types 2 and 3 as treatment…