Gene Therapy and the Global Initiative for Newborn Screening
September 26th 2023Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed factors holding back more widespread adoption of newborn screening for indications treatable with gene therapy.
Sebastian Michels, MD, on the Role of Alternative Polyadenylation in ALS Disease Pathogenesis
September 17th 2023The postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed findings he presented at ANA’s 2023 conference that may have implications for future RNA therapy development.