Children with spinal muscular atrophy (SMA) who are nonambulatory, or unable to walk, have weaker upper leg bones — as assessed by tests of bone mineral density, or BMD — compared with nonambulatory children with cerebral palsy, a group of movement disorders. Those are the results of a…
News
Throughout 2025, the team at SMA News Today has brought our audience the latest advances in spinal muscular atrophy (SMA), from emerging treatments and clinical trial updates to real-world research. As the year comes to a close, we’ve taken a look back at the top 10 stories that…
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 from Japan. Given certain limitations in the study’s design, the researchers stressed that…
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 therapeutic choice,” according to Angela Lek, PhD, chief research officer at the Muscular…
A new review of studies from six countries reveals that incorporating spinal muscular atrophy (SMA) into newborn screening (NBS) programs yields clinical improvements and reduces healthcare spending, particularly when paired with presymptomatic treatment. “These results collectively indicate that NBS for SMA, when combined with presymptomatic treatment, is not only…
Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according to a recent study. Combining mifepristone with a gene therapy construct similar to the one used in Zolgensma and Itvisma, which is intended to increase levels of…
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improved motor function in children with spinal muscular atrophy (SMA) who needed tracheostomy, without posing new safety risks, a real-world study showed. Tracheostomy is a surgical procedure that creates an opening in the windpipe, often necessary when a ventilator is used to assist…
Fatigue in the diaphragm — the large muscle at the base of the chest that pulls air in and out of the lungs — may contribute to difficulty breathing in people with spinal muscular atrophy (SMA), a new study reports. The study did not find evidence of fatigue in…
Last week’s U.S. Food and Drug Administration (FDA) approval of Novartis‘ gene therapy Itvisma (onasemnogene abeparvovec-brve) means one-time gene therapy is now authorized for all people with spinal muscular atrophy (SMA) in the U.S., an “important moment” for the SMA community, said Tracey Dawson, PhD, senior vice…
The specialty pharmacy Orsini was selected as a partner to provide Itvisma (onasemnogene abeparvovec-brve), the newly approved gene therapy for people with spinal muscular atrophy (SMA), ages 2 and older. Itvisma, marketed by Novartis, is the first gene therapy approved in the U.S. for older children and…
