Phenotypes of undiagnosed adults with actionable OTC and GLA variants

Gold JI, Madhavan S, Park J, Zouk H, Perez E, Strong A, Drivas TG, Karaa A, Yudkoff M, Rader D, Regeneron Genetics Center, Penn Medicine BioBank, Green RC, Gold NB
Human Genetics and Genomics Advances July 2023