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Biohaven Pharmaceuticals has completed the enrollment of children and young adults with spinal muscular atrophy (SMA) in a pivotal Phase 3 trial in the U.S. and Europe that is testing its experimental muscle-targeting therapy taldefgrobep alfa. The company had expected to recruit about 180 patients of all…
Children with spinal muscular atrophy (SMA) treated with Zolgensma (onasemnogene abeparvovec) used fewer healthcare resources and had lower medical costs than children on Spinraza (nusinersen), but they had higher SMA treatment costs, according to a recent U.S. analysis. Overall, healthcare costs for SMA patients using these…
Adults with spinal muscular atrophy (SMA) experience more health problems affecting a range of bodily systems compared with the general population, according to a U.S. healthcare claims analysis. A total of 30 conditions examined in the study, many of which are associated with older age in the general…
Spinraza (nusinersen) led to clinically meaningful motor improvements for three of six adults with spinal muscular atrophy (SMA) types 2 or 3 who were treated at a hospital in Japan. Other aspects, including respiratory and swallowing function, did not change considerably. “The distinct efficacy of [Spinraza] requires…
Evrysdi (risdiplam) safely stabilizes or improves motor function for most people with spinal muscular atrophy (SMA) types 1, 2, or 3, according to a new meta-analysis that reviewed more than a half-dozen studies of the approved therapy. The findings related to respiratory function were less consistent, however,…
The European Commission (EC) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old, making the therapy now available to treat SMA patients across all age ranges in Europe. Evrysdi was first approved in the European Union…
When you begin working with a patient with spinal muscular atrophy (SMA), it’s likely you will be working with them for several years. Over time, it is natural to form a bond with them, which will enable you to provide a more in-depth and more personalized treatment plan. It…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) safely and effectively treats infants with spinal muscular atrophy (SMA), especially when given within the first two months of life, according to the findings of a real-world, small study in Italy. Zolgensma “is an excellent therapeutic option for SMA patients” the…
Treatment with Spinraza (nusinersen) was found to improve psychosocial function — the day-to-day ability to contend with environmental and social tasks — in people with later-onset spinal muscular atrophy (SMA) in a new study of health-related quality of life. Importantly, patients’ abilities to communicate with their healthcare providers…
About 80% of the people living with spinal muscular atrophy (SMA) in the U.S. have used telemedicine at least once, with patients turning to it more frequently than their caregivers, according to a 2021 Cure SMA survey. SMA patients also expressed being more comfortable with telemedicine, and perceive…
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