A small molecule known as 10H-phenothiazine (PTZ) reduced the loss of motor neurons, the nerve cells lost in spinal muscular atrophy (SMA), in animal models of the disease. The treatment also rescued SMA-related changes in the structure of nerve cells, according to a study. In a mouse model, it…
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Changes in the motor unit, which is made up of a nerve cell and the muscle fibers it controls, may help doctors better understand how severe spinal muscular atrophy (SMA) is in each person, according to a new study. The researchers found that people with more severe SMA had…
Alice Wong, a disability rights activist with spinal muscular atrophy (SMA), devoted herself to fighting for the rights of people with disabilities and other underrepresented communities. When she died at age 51 in November, she left behind a legacy of advocacy and action. In her 2022 memoir, “Year…
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 needed, they said available data indicate that Evrysdi “shows a favorable…
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…
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…
