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231 results

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Page 1
Early-onset reduced bone mineral density in patients with pyruvate kinase deficiency.
Al-Samkari H, Grace RF, Glenthøj A, Andres O, Barcellini W, Galactéros F, Kuo KHM, Layton DM, Morado Arias M, Viprakasit V, Dong Y, Tai F, Hawkins P, Gheuens S, Morales-Arias J, Gilroy KS, Porter JB, van Beers EJ. Al-Samkari H, et al. Among authors: porter jb. Am J Hematol. 2023 Mar;98(3):E57-E60. doi: 10.1002/ajh.26830. Epub 2023 Jan 9. Am J Hematol. 2023. PMID: 36594181 Free article. No abstract available.
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.
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
Al-Samkari H, Galactéros F, Glenthøj A, Rothman JA, Andres O, Grace RF, Morado-Arias M, Layton DM, Onodera K, Verhovsek M, Barcellini W, Chonat S, Judge MP, Zagadailov E, Xu R, Hawkins P, Beynon V, Gheuens S, van Beers EJ; ACTIVATE Investigators. Al-Samkari H, et al. N Engl J Med. 2022 Apr 14;386(15):1432-1442. doi: 10.1056/NEJMoa2116634. N Engl J Med. 2022. PMID: 35417638 Clinical Trial.
An open-label, multicenter, efficacy, and safety study of deferasirox in iron-overloaded patients with non-transfusion-dependent thalassemia (THETIS): 5-year results.
Lai YR, Cappellini MD, Aydinok Y, Porter J, Karakas Z, Viprakasit V, Siritanaratkul N, Kattamis A, Liu R, Izquierdo M, Lasher J, Govindaraju S, Taher A. Lai YR, et al. Am J Hematol. 2022 Aug;97(8):E281-E284. doi: 10.1002/ajh.26592. Epub 2022 May 27. Am J Hematol. 2022. PMID: 35560253 Free article. No abstract available.
Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial.
Glenthøj A, van Beers EJ, Al-Samkari H, Viprakasit V, Kuo KHM, Galactéros F, Chonat S, Porter J, Zagadailov E, Xu R, Oluyadi A, Hawkins P, Gheuens S, Beynon V, Barcellini W; ACTIVATE-T investigators. Glenthøj A, et al. Lancet Haematol. 2022 Oct;9(10):e724-e732. doi: 10.1016/S2352-3026(22)00214-9. Epub 2022 Aug 18. Lancet Haematol. 2022. PMID: 35988546 Clinical Trial.
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: porter jb. 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.
Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias.
Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Cappellini MD, et al. Haematologica. 2010 Apr;95(4):557-66. doi: 10.3324/haematol.2009.014696. Epub 2009 Nov 30. Haematologica. 2010. PMID: 19951979 Free PMC article. Clinical Trial.
231 results