Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History StudyRachael F. Grace, Wilma Barcellini, Nina Kollmar et al.|Blood|2018Cited by 165
Comparison of Outcomes of Myeloablative Allogeneic Stem Cell Transplantation for Pediatric Patients with Bone Marrow Failure, Myelodysplastic Syndrome and Acute Myeloid Leukemia with and without Germline GATA2 MutationsInga Hofmann, Leslie Lehmann, Serine Avagyan et al.|Transplantation and Cellular Therapy|2020Cited by 35
Building a Harmonized Datamart by Integrating Cross-Institutional Systems of Clinical, Outcome, and Genomic Data: The Pediatric Patient Informatics Platform (<i>PPIP</i>)Clement Ma, Wendy B. London, Madhumitha Sridharan et al.|JCO Clinical Cancer Informatics|2021Cited by 5
Molecular Characterization of 140 Patients in the Pyruvate Kinase Deficiency (PKD) Natural History Study (NHS): Report of 20 New VariantsPaola Bianchi, Rachael F. Grace, Elisa Fermo et al.|Blood|2015Cited by 4
Iron Overload Is Highly Prevalent in All Disease Severity States in Pyruvate Kinase Deficiency (PKD)Eduard J. van Beers, Rachael F. Grace, Wilma Barcellini et al.|Blood|2016Cited by 3