Johns Hopkins researchers, along with academic and drug industry investigators, say they have identified a new biological target for treating spinal muscular atrophy (SMA). The scientists report they have evidence that an experimental medicine aimed at this target works as a "booster" in conjunction with a drug called nusinersen, which was recently approved by the FDA, to improve symptoms of the disorder in mice. In a report on the work, published in the January 4, 2017 issue of Neuron, the researchers say the combination therapy improved survival time, body weight and motor movements in mice with spinal muscular atrophy, a relatively rare inherited disorder characterized by the loss of motor neurons, or nerve cells that control movement. The Neuron article is titled “The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.” Nusinersen, approved by the U.S. Food and Drug Administration in December 2016, became the first drug approved to treat spinal muscular atrophy, with several other experimental drugs still in clinical trials. The Johns Hopkins researchers and their colleagues say the "boosted" nusinersen therapy, if confirmed by further studies, has the potential to significantly improve the lives of people with spinal muscular atrophy, some with forms of the disease with only weeks or months to live, and others who are unable to sit, stand or walk. The researchers caution that the booster therapy used in their new study will not be available on the market or even for use in human trials anytime soon; it must await years of animal testing for safety and effectiveness first. But the results of their study, they add, offer at least one path to extending the usefulness of nusinersen.
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