Cure 4 The Kids Foundation

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Donald B. Kohn, Claire Booth, Kit L. Shaw et al. · New England Journal of Medicine · 2021 · 218 citations · Full text

Life Sciences, Immunology, Molecular Medicine +12

Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease

James F. Casella, Bruce Barton, Julie Kanter et al. · JAMA · 2021 · 41 citations · Full text

Medicine, Hematology, Pharmacotherapy +13

Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial

Margaret V. Ragni, Scott D. Rothenberger, Robert Feldman et al. · The Lancet Haematology · 2023 · 36 citations · Full text

Medicine, Hematology, Pharmacotherapy +16

L‐leucine improves anemia and growth in patients with transfusion‐dependent Diamond‐Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond‐Blackfan Anemia Registry

Adrianna Vlachos, Evangelia Atsidaftos, Mohammad Lutfi Lababidi et al. · Pediatric Blood & Cancer · 2020 · 34 citations · Full text

Medicine, Hematology, Pharmacotherapy +13

A Phase II Study of Recombinant Interleukin-2 With or Without Recombinant Interferon-?? in Non-Hodgkin??s Lymphoma. A Study of the Cancer and Leukemia Group B

David B. Duggan, María Teresa Santarelli, Kenneth W. Zamkoff et al. · Journal of Immunotherapy · 1992 · 23 citations

Medicine, Immunology, Oncology +17