Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated with a better clinical response to Spinraza (nusinersen) among people with spinal muscular atrophy (SMA), according to a recent study in Turkey. Researchers analyzed whether levels of proteins derived from…
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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 to address the evolving needs of individuals living with SMA, and are deeply…
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 new study shows. “Combining [breathing exercises] with trunk control exercises yielded synergistic improvements…
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…
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…
