Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease. - de Castro, María José; Jones, Simon A; de las Heras, Javier; Sánchez-Pintos, Paula; Couce, María L; Colón, Cristóbal; Crujeiras, Pablo; Unceta, María; Church, Heather; Brammeier, Kathryn; Yee, Wu Hoi; Cooper, James; et al. (Orphanet Journal of Rare Diseases, 2024)