Spinal muscular atrophy (SMA) treatment with Biogen’s Spinraza (nusinersen) has resulted in motor milestone achievements in line with normal motor development and continues to provide benefit to infants who began receiving the treatment in a presymptomatic stage of the disease, according to interim clinical trial results. The study, “…
Learning that spinal muscle atrophy was the reason her 15-month-old son couldn’t do what others his age could was a “gut-wrenching” moment, Kristen Farrell recalled in a recent webcast hosted by Biogen. But, as Braeden turned 2, a clinical trial opened for a potential SMA treatment — known then only…
A video game called ACTIVE could be used to monitor changes in motor function in people with spinal muscular atrophy (SMA) types 2 and 3, according to a new study. The video game’s measurements aligned with standard tests used to monitor people with SMA, but require less expertise to administer than these…
Spinal muscular atrophy (SMA) patients transitioning from pediatric to adult care face unmet health needs and possible barriers to health services, according to a study aimed at providing insight into how adults with SMA experience the healthcare system, with a particular emphasis on their transition from pediatric to adult…
Through extensive experiments in cell and animal models of spinal muscular atrophy (SMA), investigators have demonstrated the possible usefulness of two protease inhibitors — compounds that stop certain enzymes from degrading proteins — to treat SMA, a study reports. Researchers reported that these two protease inhibitors can increase the levels…
Early intervention is crucial for the successful management of spinal muscular atrophy (SMA), according to the authors of a review study that discusses the progress made in newborn screening and treatment strategies for this disease. The review study, “Progress in treatment and newborn screening for Duchenne…