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…
A newborn screening program for spinal muscular atrophy (SMA) in Latvia can diagnose babies within two weeks of birth, according to a new study. The study, “New-Born Screening for Spinal Muscular Atrophy: Results of a Latvian Pilot Study,” was published in the International Journal of Neonatal…
Most of the children with spinal muscular atrophy (SMA) who received the gene therapy Zolgensma (onasemnogene abeparvovec) in their first weeks of life are able to walk independently within developmentally normal age ranges, according to new research from the SPR1NT clinical trial. The findings were presented at this year’s…
In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most of the seven children treated for at least one year were able to stand and walk within developmentally normal windows. These new data were presented at the Muscular Dystrophy Association…
Even when given to young children with spinal muscular atrophy (SMA) at ages older than 6 months, the gene therapy Zolgensma (onasemnogene abeparvovec) can improve motor function, an analysis of real-world data show. Indeed, more than 70% of babies treated between 6 and 24 months had better scores…
Treatment with Evrysdi (risdiplam) continues to be safe and well tolerated in people with spinal muscular atrophy (SMA) who have previously received other therapies for SMA, according to one-year data from the JEWELFISH clinical trial. Results also suggest that motor function has been stable in JEWELFISH participants after a…
