Early initiation of enzyme replacement therapy as facilitated by newborn screening improves health outcomes among patients with infantile-onset Pompe disease. - Desai AK; Rodriguez-Rassi E; Parikh S; Russo RS; Kronn D; Hamm JA; Chang IJ; Ortiz D; McPheron M; Lydigsen H; DeArmey S; Young SP; et al. (Genetics in medicine open, 2025)