The U.S. Food and Drug Administration (FDA) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old. Evrysdi, an oral therapy that works by increasing the levels of the survival motor neuron (SMN) protein that is missing in…
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…
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…
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…
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…
Treatment with the experimental muscle-directed therapy apitegromab improved motor function for many of the children and young adults with types 2 and 3 spinal muscular atrophy (SMA) after one year in the Phase 2 TOPAZ trial. Trial results were presented at the 2022 annual meeting of the American…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
Researchers in Hong Kong have shared a case report about how they were inspired and humbled by a teenage girl with spinal muscular atrophy (SMA) who got through a difficult hospital stay while maintaining a hopeful spirit and never losing sight of her goals. “Although the life of children…
Verana Health has launched a service called Neurology Qdata, which contains disease-specific datasets to help facilitate neuroscience research and develop novel treatment strategies. The new resource was announced recently at the annual meeting of the American Academy of Neurology (AAN) in Seattle, Washington. The first module to launch, called…
In children with spinal muscular atrophy (SMA) who begin treatment with Spinraza (nusinersen) after they are 2 months old, levels of a protein called cathepsin D in the fluid around the brain decline over time. This decline tends to be more pronounced in those who respond better to…
