Newborn screening urged for fatal neurological disorder

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Science  29 Jun 2018:
Vol. 360, Issue 6396, pp. 1385
DOI: 10.1126/science.360.6396.1385

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Roughly once a day in the United States, a child is born with a fatal genetic disorder that destroys motor neurons in the brain stem and spinal cord. In its worst, most common form, spinal muscular atrophy (SMA) kills children while they are toddlers, as their respiratory muscles fail. But in December 2016, the Food and Drug Administration approved a first, promising treatment for SMA: nusinersen (Spinraza), made by Biogen in Cambridge, Massachusetts. Now disease groups and members of Congress are urging Health and Human Services Secretary Alex Azar to add SMA to the panel of blood tests recommended for all newborns. Under a newborn screening law, Azar has until 8 July to make a decision. Some experts want more data from human trials, but advocates say any delay will cost lives.