Hematopoietic Stem-Cell Gene Therapy for Cerebral AdrenoleukodystrophyFlorian Eichler, David A. Williams, Christine Duncan et al.|New England Journal of Medicine|2017Cited by 592
Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow TransplantationNavneet S. Majhail, C. Frederick LeMaistre, Stephanie Farnia et al.|Biology of Blood and Marrow Transplantation|2015Cited by 409
Human Osteoclast-Poor Osteopetrosis with Hypogammaglobulinemia due to TNFRSF11A (RANK) MutationsMatteo M. Guerrini, Annalisa Frattini, Cristina Sobacchi et al.|The American Journal of Human Genetics|2008Cited by 307
Hematologic Cancer after Gene Therapy for Cerebral AdrenoleukodystrophyChristine Duncan, David A. Williams, Jacob R. Bledsoe et al.|New England Journal of Medicine|2024Cited by 119
Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidanceMehmet Umut Akyol, Maurizio Scarpa, Tord D. Alden et al.|Orphanet Journal of Rare Diseases|2019Cited by 114