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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…

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…

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…

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…

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…