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576 results

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Page 1
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. Among authors: cappellini md. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.
Cappellini MD, Viprakasit V, Taher AT, Georgiev P, Kuo KHM, Coates T, Voskaridou E, Liew HK, Pazgal-Kobrowski I, Forni GL, Perrotta S, Khelif A, Lal A, Kattamis A, Vlachaki E, Origa R, Aydinok Y, Bejaoui M, Ho PJ, Chew LP, Bee PC, Lim SM, Lu MY, Tantiworawit A, Ganeva P, Gercheva L, Shah F, Neufeld EJ, Thompson A, Laadem A, Shetty JK, Zou J, Zhang J, Miteva D, Zinger T, Linde PG, Sherman ML, Hermine O, Porter J, Piga A; BELIEVE Investigators. Cappellini MD, et al. N Engl J Med. 2020 Mar 26;382(13):1219-1231. doi: 10.1056/NEJMoa1910182. N Engl J Med. 2020. PMID: 32212518 Clinical Trial.
Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with nontransfusion-dependent thalassemia syndromes.
Taher AT, Porter JB, Kattamis A, Viprakasit V, Cappellini MD. Taher AT, et al. Among authors: cappellini md. Drug Des Devel Ther. 2016 Dec 15;10:4073-4078. doi: 10.2147/DDDT.S117080. eCollection 2016. Drug Des Devel Ther. 2016. PMID: 28008230 Free PMC article. No abstract available.
β-Thalassemias. Reply.
Taher AT, Musallam KM, Cappellini MD. Taher AT, et al. Among authors: cappellini md. N Engl J Med. 2021 Jun 3;384(22):2166. doi: 10.1056/NEJMc2105064. N Engl J Med. 2021. PMID: 34077657 No abstract available.
β-Thalassemias.
Taher AT, Musallam KM, Cappellini MD. Taher AT, et al. Among authors: cappellini md. N Engl J Med. 2021 Feb 25;384(8):727-743. doi: 10.1056/NEJMra2021838. N Engl J Med. 2021. PMID: 33626255 Review. No abstract available.
Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study.
Taher AT, Cappellini MD, Aydinok Y, Porter JB, Karakas Z, Viprakasit V, Siritanaratkul N, Kattamis A, Wang C, Zhu Z, Joaquin V, Uwamahoro MJ, Lai YR. Taher AT, et al. Among authors: cappellini md. Blood Cells Mol Dis. 2016 Mar;57:23-9. doi: 10.1016/j.bcmd.2015.11.002. Epub 2015 Nov 11. Blood Cells Mol Dis. 2016. PMID: 26852651 Free article. Clinical Trial.
Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up.
Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y. Cappellini MD, et al. Blood. 2011 Jul 28;118(4):884-93. doi: 10.1182/blood-2010-11-316646. Epub 2011 May 31. Blood. 2011. PMID: 21628399 Free article. Clinical Trial.
576 results