In this Wednesday, Nov. 12, 2008 file photo, a baby cries in its bed in a hospital in Bremen, northern Germany. (AP Photo/Joerg Sarbach)
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Only 15 babies with spinal muscular atrophy received the experimental gene therapy, but researchers in Ohio credited the preliminary and promising results to replacing the infants' defective gene early -- in the first few months of life, before the neuromuscular disease destroyed too many key nerve cells.Mendell cautioned that much more study is needed to prove the gene therapy works and is safe.Three babies received a low dose of the gene therapy, as a first-step safety precaution.All of the children are alive, Mendell said, about two years and counting after treatment. The only serious side effect attributed to the gene therapy so far involved possible signs of a liver problem that eased with treatment.AveXis Inc., which is developing the gene therapy and helped fund Wednesday's study, has opened a second small trial at seven hospitals.
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