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214 results

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Page 1
Diagnosis and management of pyruvate kinase deficiency: international expert guidelines.
Al-Samkari H, Shehata N, Lang-Robertson K, Bianchi P, Glenthøj A, Sheth S, Neufeld EJ, Rees DC, Chonat S, Kuo KHM, Rothman JA, Barcellini W, van Beers EJ, Pospíšilová D, Shah AJ, van Wijk R, Glader B, Mañú Pereira MDM, Andres O, Kalfa TA, Eber SW, Gallagher PG, Kwiatkowski JL, Galacteros F, Lander C, Watson A, Elbard R, Peereboom D, Grace RF. Al-Samkari H, et al. Among authors: neufeld ej. Lancet Haematol. 2024 Mar;11(3):e228-e239. doi: 10.1016/S2352-3026(23)00377-0. Epub 2024 Feb 5. Lancet Haematol. 2024. PMID: 38330977 Review.
Dysregulated arginine metabolism and cardiopulmonary dysfunction in patients with thalassaemia.
Morris CR, Kim HY, Klings ES, Wood J, Porter JB, Trachtenberg F, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Hassell K, Taher A, Neufeld EJ, Thompson AA, Larkin S, Suh JH, Vichinsky EP, Kuypers FA; Thalassemia Clinical Research Network. Morris CR, et al. Among authors: neufeld ej. Br J Haematol. 2015 Jun;169(6):887-98. doi: 10.1111/bjh.13452. Epub 2015 Apr 24. Br J Haematol. 2015. PMID: 25907665 Free PMC article.
Genetic variants in canonical Wnt signaling pathway associated with pediatric immune thrombocytopenia.
Kim TO, Geris JM, Flanagan JM, Grace RF, Lambert MP, O'Farrell C, Rose MJ, Shimano KA, Niss O, Neunert C, Nakano TA, MacMath D, Dinu B, Kirk SE, Neufeld EJ, Despotovic JM, Scheurer ME, Grimes AB. Kim TO, et al. Among authors: neufeld ej. Blood Adv. 2024 Nov 12;8(21):5529-5538. doi: 10.1182/bloodadvances.2024012776. Blood Adv. 2024. PMID: 39189922 Free PMC article.
Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study.
Mathias M, Abraham A, Belletrutti MJ, Carcao M, Carvalho M, Chambost H, Chan AKC, Dubey L, Ducore J, Gattens M, Gresele P, Gruel Y, Guillet B, Jiménez-Yuste V, Kitanovski L, Klukowska A, Lohade S, Mancuso ME, Oldenburg J, Pollio B, Sigaud M, Vilchevska K, Wu JKM, Jansen M, Belyanskaya L, Walter O, Knaub S, Neufeld EJ. Mathias M, et al. Among authors: neufeld ej. Eur J Haematol. 2023 Oct;111(4):544-552. doi: 10.1111/ejh.14040. Epub 2023 Jul 13. Eur J Haematol. 2023. PMID: 37439123
Health-related quality of life in patients with β-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept.
Cappellini MD, Taher AT, Piga A, Shah F, Voskaridou E, Viprakasit V, Porter JB, Hermine O, Neufeld EJ, Thompson AA, Tang D, Yucel A, Lord-Bessen J, Yu P, Guo S, Shetty JK, Miteva D, Zinger T, Backstrom JT, Oliva EN. Cappellini MD, et al. Among authors: neufeld ej. Eur J Haematol. 2023 Jul;111(1):113-124. doi: 10.1111/ejh.13975. Epub 2023 Apr 24. Eur J Haematol. 2023. PMID: 37095595 Clinical Trial.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding.
Ragni MV, Young G, Batsuli G, Bisson E, Carpenter SL, Croteau SE, Cuker A, Curtis RG, Denne M, Ewenstein B, Federizo A, Frick N, Funkhouser K, George LA, Hoots WK, Jobe SM, Krava E, Langmead CJ, Lewis RJ, López J, Malec L, Mann Z, Miles ME 3rd, Neely E, Neufeld EJ, Pierce GF, Pipe SW, Pitler LR, Raffini L, Schnur KM, Shavit JA. Ragni MV, et al. Among authors: neufeld ej. Expert Rev Hematol. 2023 Mar;16(sup1):107-127. doi: 10.1080/17474086.2023.2181781. Expert Rev Hematol. 2023. PMID: 36920855
Phase 3 Trial of Sotatercept for Treatment of Pulmonary Arterial Hypertension.
Hoeper MM, Badesch DB, Ghofrani HA, Gibbs JSR, Gomberg-Maitland M, McLaughlin VV, Preston IR, Souza R, Waxman AB, Grünig E, Kopeć G, Meyer G, Olsson KM, Rosenkranz S, Xu Y, Miller B, Fowler M, Butler J, Koglin J, de Oliveira Pena J, Humbert M; STELLAR Trial Investigators. Hoeper MM, et al. N Engl J Med. 2023 Apr 20;388(16):1478-1490. doi: 10.1056/NEJMoa2213558. Epub 2023 Mar 6. N Engl J Med. 2023. PMID: 36877098 Clinical Trial.
Gene Therapy and Hemophilia: Where Do We Go from Here?
Bolous NS, Bhatt N, Bhakta N, Neufeld EJ, Davidoff AM, Reiss UM. Bolous NS, et al. Among authors: neufeld ej. J Blood Med. 2022 Oct 6;13:559-580. doi: 10.2147/JBM.S371438. eCollection 2022. J Blood Med. 2022. PMID: 36226233 Free PMC article. Review.
214 results