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…
