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121 results

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Page 1
Gene addition for beta thalassemia.
Kwiatkowski JL. Kwiatkowski JL. Ann N Y Acad Sci. 2023 Dec;1530(1):105-109. doi: 10.1111/nyas.15070. Epub 2023 Oct 13. Ann N Y Acad Sci. 2023. PMID: 37828865 Review.
Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open-label FIRST study.
Hamdy M, El-Beshlawy A, Veríssimo MPA, Kanter J, Inusa B, Williams S, Lee D, Temin NT, Fradette C, Tricta F, Ebeid FSE, Kwiatkowski JL, Elalfy MS. Hamdy M, et al. Among authors: kwiatkowski jl. Pediatr Blood Cancer. 2024 Jan;71(1):e30711. doi: 10.1002/pbc.30711. Epub 2023 Oct 9. Pediatr Blood Cancer. 2024. PMID: 37807937 Clinical Trial.
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
Kwiatkowski JL, Walters MC, Hongeng S, Yannaki E, Kulozik AE, Kunz JB, Sauer MG, Thrasher AJ, Thuret I, Lal A, Tao G, Ali S, Thakar HL, Elliot H, Lodaya A, Lee J, Colvin RA, Locatelli F, Thompson AA. Kwiatkowski JL, et al. Lancet. 2024 Nov 30;404(10468):2175-2186. doi: 10.1016/S0140-6736(24)01884-1. Epub 2024 Nov 8. Lancet. 2024. PMID: 39527960 Clinical Trial.
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia.
Locatelli F, Lang P, Wall D, Meisel R, Corbacioglu S, Li AM, de la Fuente J, Shah AJ, Carpenter B, Kwiatkowski JL, Mapara M, Liem RI, Cappellini MD, Algeri M, Kattamis A, Sheth S, Grupp S, Handgretinger R, Kohli P, Shi D, Ross L, Bobruff Y, Simard C, Zhang L, Morrow PK, Hobbs WE, Frangoul H; CLIMB THAL-111 Study Group. Locatelli F, et al. Among authors: kwiatkowski jl. N Engl J Med. 2024 May 9;390(18):1663-1676. doi: 10.1056/NEJMoa2309673. Epub 2024 Apr 24. N Engl J Med. 2024. PMID: 38657265 Clinical Trial.
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: kwiatkowski jl. 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.
Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years.
Elalfy MS, Hamdy M, El-Beshlawy A, Ebeid FSE, Badr M, Kanter J, Inusa B, Adly AAM, Williams S, Kilinc Y, Lee D, Fradette C, Rozova A, Temin NT, Tricta F, Kwiatkowski JL. Elalfy MS, et al. Among authors: kwiatkowski jl. Blood Adv. 2023 Feb 28;7(4):611-619. doi: 10.1182/bloodadvances.2021006778. Blood Adv. 2023. PMID: 36018224 Free PMC article. Clinical Trial.
121 results