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103 results

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Page 1
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.
Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J; HOPE Trial Investigators. Vichinsky E, et al. N Engl J Med. 2019 Aug 8;381(6):509-519. doi: 10.1056/NEJMoa1903212. Epub 2019 Jun 14. N Engl J Med. 2019. PMID: 31199090 Clinical Trial.
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.
Piel FB, Rees DC, DeBaun MR, Nnodu O, Ranque B, Thompson AA, Ware RE, Abboud MR, Abraham A, Ambrose EE, Andemariam B, Colah R, Colombatti R, Conran N, Costa FF, Cronin RM, de Montalembert M, Elion J, Esrick E, Greenway AL, Idris IM, Issom DZ, Jain D, Jordan LC, Kaplan ZS, King AA, Lloyd-Puryear M, Oppong SA, Sharma A, Sung L, Tshilolo L, Wilkie DJ, Ohene-Frempong K. Piel FB, et al. Among authors: colombatti r. Lancet Haematol. 2023 Aug;10(8):e633-e686. doi: 10.1016/S2352-3026(23)00096-0. Epub 2023 Jul 11. Lancet Haematol. 2023. PMID: 37451304 Free PMC article. Review.
Impact of sickle cell disease on patients' daily lives, symptoms reported, and disease management strategies: Results from the international Sickle Cell World Assessment Survey (SWAY).
Osunkwo I, Andemariam B, Minniti CP, Inusa BPD, El Rassi F, Francis-Gibson B, Nero A, Trimnell C, Abboud MR, Arlet JB, Colombatti R, de Montalembert M, Jain S, Jastaniah W, Nur E, Pita M, DeBonnett L, Ramscar N, Bailey T, Rajkovic-Hooley O, James J. Osunkwo I, et al. Among authors: colombatti r. Am J Hematol. 2021 Apr 1;96(4):404-417. doi: 10.1002/ajh.26063. Epub 2021 Feb 25. Am J Hematol. 2021. PMID: 33264445 Free PMC article.
COL4A1 and COL4A2-related disorders: Clinical features, diagnostic guidelines, and management.
Tambala D, Vassar R, Snow J, Balestrini S, Bersano A, Guey S, Bonaventura E, Signorini S, Sartori S, Bertini E, Tonduti D, Parazzini C, Macchiaiolo M, Pelizza MF, Pichiecchio A, Massella L, Coste T, Orcesi S, Politano D, Bacci G, Marziali E, Dollfus H, Mandelli A, Chinali M, Plaisier E, Simioni P, Colombatti R, Guerrini R, Tournier-Lasserve E, Gould DB, Musolino PL. Tambala D, et al. Among authors: colombatti r. Genet Med. 2025 Sep;27(9):101514. doi: 10.1016/j.gim.2025.101514. Epub 2025 Jul 2. Genet Med. 2025. PMID: 40616396 Free article.
Sickle cell disease landscape and challenges in the EU: the ERN-EuroBloodNet perspective.
Mañú Pereira MDM, Colombatti R, Alvarez F, Bartolucci P, Bento C, Brunetta AL, Cela E, Christou S, Collado A, de Montalembert M, Dedeken L, Fenaux P, Galacteros F, Glenthøj A, Gutiérrez Valle V, Kattamis A, Kunz J, Lobitz S, McMahon C, Pellegrini M, Reidel S, Russo G, Santos Freire M, van Beers E, Kountouris P, Gulbis B. Mañú Pereira MDM, et al. Among authors: colombatti r. Lancet Haematol. 2023 Aug;10(8):e687-e694. doi: 10.1016/S2352-3026(23)00182-5. Epub 2023 Jul 11. Lancet Haematol. 2023. PMID: 37451300 Review.
Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial.
Idowu M, Otieno L, Dumitriu B, Lobo CLC, Thein SL, Andemariam B, Nnodu OE, Inati A, Glaros AK, Bartolucci P, Colombatti R, Taher AT, Abboud MR, Darbari D, Ataga KI, Antmen AB, Kuo KHM, de Souza Medina S, Oluyadi A, Iyer V, Morris S, Yates AM, Shao H, Patil S, Urbstonaitis R, Zaidi AU, Gheuens S, Smith WR. Idowu M, et al. Among authors: colombatti r. Lancet Haematol. 2025 Jan;12(1):e35-e44. doi: 10.1016/S2352-3026(24)00319-3. Epub 2024 Dec 4. Lancet Haematol. 2025. PMID: 39644907 Clinical Trial.
Anemia and transfusion in the neonate.
Colombatti R, Sainati L, Trevisanuto D. Colombatti R, et al. Semin Fetal Neonatal Med. 2016 Feb;21(1):2-9. doi: 10.1016/j.siny.2015.12.001. Epub 2015 Dec 28. Semin Fetal Neonatal Med. 2016. PMID: 26732078 Review.
What's wrong with drug development for sickle cell disease?
Brousse V, Rees D, Colombatti R. Brousse V, et al. Among authors: colombatti r. Hemasphere. 2025 Feb 6;9(2):e70082. doi: 10.1002/hem3.70082. eCollection 2025 Feb. Hemasphere. 2025. PMID: 39917054 Free PMC article. No abstract available.
103 results