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

Ke Q, Zhao ZY, Mendell JR, et al. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy. World journal of pediatrics : WJP 2019;15:219-225. Abstract 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 […]