Within a week of Christmas day, a drug called nusinersen (Spinraza) will be in the hands of doctors across the nation, who will use it, most urgently, to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). The leading genetic cause of infant mortality, SMA is a motor neuron disease that leads to the wasting of young muscles, impairing the ability of newborns and toddlers to walk, crawl, or even hold their heads up, and in the most severe cases, failure of muscles that enable them to breathe. Nusinersen (to be sold by Biogen under the brand name Spinraza) was conceived and tested over several years in mouse models of SMA by Professor Adrian Krainer, Ph.D., and his colleagues at Cold Spring Harbor Laboratory (CSHL) in collaboration with drug developers led by Dr. Frank Bennett at Ionis Pharmaceuticals. Their collaboration began a dozen years ago. “Some are understandably calling the FDA’s announcement of nusinersen’s approval ‘a Christmas surprise,’” Dr. Krainer commented. “For those of us who have had the thrilling experience of working on this drug from the very beginning, and have watched it succeed in reversing SMA pathology in animals—and more recently in young people with the illness—news of the approval is incredibly gratifying. Most gratifying to me is the thought that thousands of families will now be able to see their loved ones benefit from the drug’s therapeutic effects.” “This drug will save lives of young people with severe SMA, and will improve the lives of many thousands of older children and adults who have disabling forms of the disease,” said Bruce Stillman, Ph.D., President and CEO of CSHL.
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