Researchers pinpoint new target for spinal muscular atrophy
New data from a study conducted on mice has shed light on a potential new “booster” for a recently approved therapy for patients with spinal muscular atrophy.

According to findings published in Neuron, an experimental drug successfully amplified the effects of an FDA-approved drug called nusinersen, which treats the symptoms of spinal muscular atrophy.

In patients with spinal muscular atrophy, a genetic mutation of the SMN1 gene results in insufficient survival motor neuron (SMN) protein production in neurons. The findings published by Sumner and her colleagues identified lnc-RNA as a potential target for ameliorating this shortage.