Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy

Background Advances in treatment for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) hold promise for children with these disorders. Accurate genetic diagnosis, early in the disease process, will allow these treatments to be most effective. Newborn screening (NBS) for SMA has bee...

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