Author Archives: Marisa Wexler, MS

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…

Last month’s approval of Novartis‘ one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) for people with spinal muscular atrophy (SMA) ages 2 and older is a milestone that “ushers in a new era of informed…

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent apitegromab as a treatment for spinal muscular atrophy (SMA). That resubmission to the FDA is expected in…

The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new research done in laboratory models. “We demonstrated that a daily oral administration of BIO101 can specifically improve…

Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may stabilize respiratory…