Author Archives: Marisa Wexler, MS

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,…

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…

A rapid test using zebrafish was able to show that two children identified by genetic testing as possibly having spinal muscular atrophy (SMA) did not actually have the progressive disease, according to a new study.

Treatment with gene therapy significantly prolonged survival and improved motor function in a mouse model of spinal muscular atrophy with respiratory distress type 1 (SMARD1), researchers report. The gene therapy tested in this lab…

For babies diagnosed with spinal muscular atrophy (SMA) before birth, doctors may be able to plan an early delivery so the baby can start treatment within days instead of waiting for a full-term birth. In…

Spinal muscular atrophy (SMA) affects the nervous system — essentially, the body’s command center, where nerves transmit signals that coordinate actions, senses, and bodily functions — more profoundly than previously thought, a new study suggests.

The European Commission has approved a new high-dose regimen of Spinraza (nusinersen) for people with 5q spinal muscular atrophy (SMA). “We are proud to introduce the high dose regimen of SPINRAZA, which we have developed…

A program that aims to simultaneously strengthen muscles needed for breathing and postural muscles in the trunk may benefit people with spinal muscular atrophy (SMA) more than a program that focuses only on breathing muscles, a…

Evrysdi (risdiplam) can stabilize motor function, improve swallowing ability, ease fatigue, and improve life quality in adults with spinal muscular atrophy (SMA), according to a review of scientific literature. Although the researchers stressed that more studies are…

A medicine called leuprorelin acetate, approved in the U.S. to treat prostate cancer, can slow the progression of physical disability in people with spinal and bulbar muscular atrophy (SBMA), according to an analysis of a real-world study…