Gene therapy is saving children’s lives—but screening is way behind
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Some background: 15 children with spinal muscular atrophy (SMA), the leading genetic cause of infant death, have likely been saved
in a clinical trial of a gene therapy created by AveXis. The most
dramatic results are being seen in children who are treated in the first
month or two of life.
However: States have been slow to adopt screening tests for genetic diseases that are, increasingly, treatable. Newborn screening tests in the US cover a minimum of 34 disorders, and many states have opted to add more, but most don’t look for SMA.
More testing needed: Although a national committee has voted to recommend that SMA be added to the recommended universal screening panel, it’s at best an incomplete solution, as it’s non-binding in most states.
However: States have been slow to adopt screening tests for genetic diseases that are, increasingly, treatable. Newborn screening tests in the US cover a minimum of 34 disorders, and many states have opted to add more, but most don’t look for SMA.
More testing needed: Although a national committee has voted to recommend that SMA be added to the recommended universal screening panel, it’s at best an incomplete solution, as it’s non-binding in most states.
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