Encouraging news from Isis Pharmaceuticals, Inc., presented at the 19th International World Muscle Society Congress in Berlin, demonstrated the efficacy of ISIS-SMNRx in treating spinal muscular atrophy (SMA) in infants and children. Results were from the ongoing open-label Phase 2 clinical trial. Isis is also enrolling patients in the…
News
Two recent research projects involving studies into Spinal Muscular Atrophy are shedding new light on the deadly condition, which could in turn lead to viable next-generation therapies. The first new study, entitled, “SMA-causing missense mutations in survival motor neuron (Smn) display a wide range of phenotypes when modeled…
Loss of motor function, a short lifespan, and high mortality rate make spinal muscular atrophy a rare yet deadly disease for infants and children. Combined with the fact that no viable FDA-approved treatment currently exists for the disease, SMA has one of the most dire unmet medical needs in healthcare…
A recent study entitled “iPSC-Derived Neural Stem Cells Act via Kinase Inhibition to Exert Neuroprotective Effects in Spinal Muscular Atrophy with Respiratory Distress Type 1” published in Stem Cell Reports suggests therapeutic potential of neural stem cells (NSCs) from human-induced pluripotent stem cells (iPSCs) to…
In a recent study entitled “Stem cell-derived motor neurons from spinal and bulbar muscular atrophy patients,” the authors characterized stem cells and motor neuron derivatives from patients with spinal and bulbar muscular atrophy. The study, which was published in the Neurobiology of Disease journal, contributes…
Lydia Peters cutting the ribbon during the playground’s opening ceremony. Finally, parents of children with special needs can breathe easier when their little ones ask for an afternoon at the playground. It was last August 22, when over a hundred locals and public figures gathered to witness the grand…
Spinal muscular atrophy is a disease characterized by a short lifespan and progressive, debilitating symptoms. New research on mice, however, suggests that scientists may in the early stages of developing a new treatment that could eventually extend survival rates in children with SMA. A new study entitled “…
Parents want what is best for their children, and parents of children with spinal muscular atrophy (SMA) are no exception. At times, these parents can feel stressed about their children’s medical condition, and any way that stress can be alleviated is vital to improving parent outlook on managing their children’s…
