The Growing Case For Screening Newborns
Fatal Disorder Can Be Treated With Cord-Blood Transplant If Caught at Birth, Study Says
By DAVID ARMSTRONG
Staff Reporter of THE WALL STREET JOURNAL
May 19, 2005; Page D1
A new study gives added weight to the growing movement to test newborn babies for an array of rare but life-threatening genetic disorders. In the study, researchers from Duke University and the University of North Carolina found that a transplant of donor umbilical-cord blood soon after birth can save babies with Krabbe disease, an often-fatal enzyme illness. Of 25 babies treated, 18 were saved by the procedure.
No states test newborns for Krabbe or most of the other 44 enzyme conditions in a class known as lysosomal-storage disorders -- in part because the cases have long been considered hopeless. Babies born with Krabbe disease, which can impair development and cause seizures, usually die before reaching two years of age if untreated. The study, published in this week's New England Journal of Medicine, is the latest evidence that at least some lysosomal diseases can be treated successfully, especially if caught at birth.
The research gives a significant boost to efforts by patient-advocacy groups and many physicians to create a universal standard for newborn screenings that would apply to all states and include far more diseases than most states now test for. Currently, requirements vary widely from state to state, with some testing for as few as three conditions and others screening for as many as 43, ranging from hypothyroidism to sickle-cell disease. As a result, advocates for universal screening say the fate of babies with genetic problems can depend simply on where they were born.
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