News

Zolgensma into spinal canal drives SMA type 2 motor gains

Treatment with OAV101 IT, a formulation of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s delivered directly into the spinal canal, safely led to motor improvements for children with spinal muscular atrophy (SMA) type 2 who were at least 2 years old,  top-line results from the Phase 3 STEER…

Top 10 SMA news stories of 2024

Throughout 2024, SMA News Today brought our readers daily coverage of the latest clinical research and scientific breakthroughs related to spinal muscular atrophy (SMA). Here are the top 10 most-read stories we published last year, each with a brief description. We’re excited to continue serving the SMA community in…

Model predicts scoliosis risk in patients receiving SMA treatment

An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height may help predict the risk of scoliosis in people with spinal muscular atrophy (SMA) who have received disease-modifying treatment. “We trained a scoliosis classifier, demonstrating how data give rise…

Swallowing problems in SMA may be declining since advent of DMTs

Swallowing function may have improved in spinal muscular atrophy (SMA) patients since disease-modifying therapies (DMT) have become available, a review study suggests. The analysis showed problems with swallowing were prevalent in the years before the emergence of DMTs and that studies after the treatments became available indicated stabilizations…

Hand strength improves for adults with SMA types 2, 3 taking Evrysdi

Evrysdi (risdiplam) effectively improved motor function in adults with spinal muscular atrophy (SMA) type 2 or type 3, particularly skills like hand strength and function, a small study in France shows. Most patients also reported gains in bulbar functions, that is, speaking, chewing, and swallowing, and eased breath…