News

A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with spinal muscular atrophy (SMA).  CADTH is an independent, nonprofit organization that provides evidence-based advice to…

Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an analysis of safety data from the U.S.

Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier group. Notably, 8.5 kilograms was the maximum weight of SMA patients enrolled in clinical trials supporting Zolgensma’s approval.

From being a child actor to a lawyer in the entertainment industry, Alexa Dectis, who has relied on a power wheelchair to get around since she was 2, has gone far. Dectis, diagnosed with spinal muscular atrophy (SMA) type 2 when she was 18 months old, was named…

Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy (SMA) types 2 or 3, according to a study from Israel. The study, “Longer-term follow-up of nusinersen efficacy and safety in adult patients with spinal muscular atrophy…

A second-generation investigational gene therapy for spinal muscular atrophy (SMA) showed greater safety and efficacy than an earlier version, and it may be more effective than the currently approved gene therapy for SMA, a study in a disease mouse model reported. The study was conducted by scientists with CANbridge…

For parents, protecting the health and well-being of a child with spinal muscular atrophy (SMA) was a top priority when the COVID-19 pandemic first hit the Netherlands in early 2020, despite the difficulties isolation can bring, an interview study reports. The study, “‘This battle, between your…

The stability and self-interacting ability of SMN, the protein that is abnormally low in people with spinal muscular atrophy (SMA), may affect disease severity in rare cases, a small study suggested. Based on their findings, the researchers said new treatment strategies “should be explored” to target these factors, and…

Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of life are continuing to gain motor skills after three years of treatment, according to new data from the FIREFISH trial. “These long-term results in babies treated with Evrysdi are very…

A surgeon at UC Davis Health has successfully performed the first hip replacement in a person — in this case, a 16-year-old girl — with spinal muscular atrophy (SMA). Before the surgery, the girl, named Malena, had pain and limited movement caused by arthritis that had developed between…