A combination of approved treatments, alongside early physical therapy, was successfully deployed to help treat a boy with spinal muscular atrophy (SMA) type 0 in Japan. Type 0 is the most severe form of SMA, marked by symptoms that start in the womb prior to birth. Without…
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Even when treated early with disease-modifying therapies (DMTs), infants and toddlers with spinal muscular atrophy (SMA) do not develop swallowing skills at the same pace or to the same level as healthy children. That’s according to a new study from Germany, which tracked more than 30 children with…
Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a letter to the SMA community, this “difficult decision” follows results from the first…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results show. These findings come from the STEER Phase 3 trial (NCT05089656), which supported…
Up to three years of treatment with Evrysdi (risdiplam) appears safe and was associated with presymptomatic children with spinal muscular atrophy (SMA) reaching and maintaining key developmental milestones, according to Phase 2 study results. “The majority of children treated with [Evrysdi] before the onset of SMA symptoms, maintained…
A high-dose regimen of Spinraza (nusinersen) that is up for regulatory approval in the U.S. may help stabilize or improve motor function for people with spinal muscular atrophy (SMA), pooled results from two clinical trials show. “Overall, the improvements observed exceeded what would be expected in a broad…
As a licensed clinical social worker based in Amarillo, Texas, Ali Ramos uses her own experience of living with spinal muscular atrophy (SMA) type 2 to create a safe space for her clients to explore their feelings. Her personal journey informs the mental health care and advocacy she…
Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in children with SMA who had previously been treated with gene therapy but did not respond as…
Mutations that cause spinal muscular atrophy (SMA) disrupt the development of spinal cord cells, leading to fewer nerve cells in early development, according to a new study in cell models. The findings highlight potential new avenues for treating SMA in its earliest stages, researchers say. Zeynep Dokuzluoglu, a graduate…
