Jul 29, 2020 · BACKGROUND AND OBJECTIVES: Historically, autosomal recessive 5q-linked spinal muscular atrophy (SMA) has been the leading inherited cause of infant death. SMA is caused by the absence of the SMN1 gene, and SMN1 gene replacement therapy, onasemnogene abeparvovec-xioi, was Food and Drug Administration approved in May 2019. Approval included all children with SMA age <2 years without end-stage ...
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    Congratulations to Andrew McCutchen, winner of the 2015 Roberto Clemente Award:. McCutchen founded “Cutch’s Crew” in 2010 to assist inner-city youth baseball players and at-risk children in Pittsburgh.

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