Baby gene therapy study offers hope for fatal muscle disease

A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots' lives, and some began to roll over, sit and stand on their own, researchers reported Wednesday. 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. "They all should have died by now," said Dr. Jerry Mendell of Nationwide Children's...

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