HIGHLIGHTS
- who: Richard B. Parad et al. from the Department of Pediatric Medicine, Brigham and Women's, Harvard Medical School, Inc, Raleigh, NC, USA have published the article: Implementation of Hospital-Based Supplemental Duchenne Muscular Dystrophy Newborn Screening (sDMDNBS): A Pathway to Broadening Adoption, in the Journal: (JOURNAL)
- what: Although this approach has not yet been attempted in humans, animal studies suggest that MyoAAVs largely avoid the liver, raising the prospect for more effective gene therapies without the risk of liver damage and other serious side effects . Because the group had previously developed NBS-specific requirements . . .
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