The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was safe and effective in a baby girl with spinal muscular atrophy (SMA) type 1 who was previously on Evrysdi (risdiplam) for about three months, a case study reports. This likely represents the first report of safety and efficacy outcomes of…

Disease-modifying therapies (DMTs) used for spinal muscular atrophy (SMA) may contribute to the development of therapies for age-related progressive loss of muscle mass and strength, or sarcopenia, new research suggests. The survival muscle neuron (SMN) protein, whose deficiency causes SMA, was found at progressively lower levels with aging in…

With all due gravity, I informed the group chat about my latest hypothesis. “I think I might be lactose intolerant.” “NO,” said one friend. “NOOOOO,” said another. I explained that I’d been experiencing some strange symptoms around dinnertime. Initially, I assumed it had something to do with Evrysdi (risdiplam),…

Damn Taylor Swift and her lyricism. I had a different plan for this column, with a different title in mind for it. I was going to ramble about moving out of the condominium I’ve been staying in for almost two years. There was to be a farewell to its inaccessible…

After two years of treatment with Evrysdi (risdiplam), most babies with spinal muscular atrophy (SMA) type 1 in the FIREFISH clinical trial are still alive without a need for permanent ventilation, and many of the youngsters are showing continual improvements in motor development. The results were published in …

Continued treatment with apitegromab, designed to strengthen muscles, led to stabilization or improvement in measures of fatigue and life quality for children and young adults with types 2 and 3 spinal muscular atrophy (SMA), according to new data from the TOPAZ clinical trial. According to apitegromab’s developer…

Children with spinal muscular atrophy (SMA) switching from Spinraza (nusinersen) to Evrysdi (risdiplam) are generally optimistic about the switch as are their caregivers, according to a new study. The report, “How children and caregivers viewed the change from nusinersen to risdiplam for treating spinal…

Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial that enrolled spinal muscular atrophy (SMA) patients who had previously been on other therapies, two-year data show. “These important data demonstrate the safety and efficacy of Evrysdi in a…

Content warning: The following contains mentions of medical trauma and death. I knew something was wrong. But I didn’t always. When my friend and fellow columnist Sherry Toh messaged me one evening to say she was sick, I assumed it was a cold. She would disappear for a few days…

Doctors need to be well-educated about options in the evolving treatment landscape to help patients with spinal muscular atrophy (SMA) make informed decisions, say neurologists in a panel discussion. The panel of four neurologists with experience in treating SMA patients was convened by Biogen, which markets…