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Page 1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. Among authors: crary se. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators. Coppens M, et al. Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1. Lancet Haematol. 2024. PMID: 38437857 Clinical Trial.
Anticoagulation and vascular anomalies.
Crary SE, Mack JM. Crary SE, et al. Res Pract Thromb Haemost. 2024 Apr 3;8(3):102402. doi: 10.1016/j.rpth.2024.102402. eCollection 2024 Mar. Res Pract Thromb Haemost. 2024. PMID: 38694837 Free PMC article.
Barriers to Genetic Testing in Vascular Malformations.
Borst AJ, Hammill AM, Crary SE, McLean TW, Felton T, Blatt J. Borst AJ, et al. Among authors: crary se. JAMA Netw Open. 2023 May 1;6(5):e2314829. doi: 10.1001/jamanetworkopen.2023.14829. JAMA Netw Open. 2023. PMID: 37219903 Free PMC article.
Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study.
Borst AJ, Eng W, Griffin M, Ricci KW, Engel E, Adams DM, Dayneka J, Cohen-Cutler SJ, Andreoli SM, Wu MD, Wheeler AP, Heym KM, Crary SE, Nakano TA, Schulte RR, Setty BA, McLean TW, Pahl KS, Intzes S, Pateva I, Teitelbaum M, Zong Z, Li Y, Jeng MR. Borst AJ, et al. Among authors: crary se. Pediatr Blood Cancer. 2024 Mar;71(3):e30779. doi: 10.1002/pbc.30779. Epub 2023 Dec 10. Pediatr Blood Cancer. 2024. PMID: 38073018
How we approach coagulopathy with vascular anomalies.
Mack JM, Crary SE. Mack JM, et al. Among authors: crary se. Pediatr Blood Cancer. 2022 Aug;69 Suppl 3:e29353. doi: 10.1002/pbc.29353. Pediatr Blood Cancer. 2022. PMID: 36070211 Review.
Effect of Anticoagulant Therapy for 6 Weeks vs 3 Months on Recurrence and Bleeding Events in Patients Younger Than 21 Years of Age With Provoked Venous Thromboembolism: The Kids-DOTT Randomized Clinical Trial.
Goldenberg NA, Kittelson JM, Abshire TC, Bonaca M, Casella JF, Dale RA, Halperin JL, Hamblin F, Kessler CM, Manco-Johnson MJ, Sidonio RF, Spyropoulos AC, Steg PG, Turpie AGG, Schulman S; Kids-DOTT Trial Investigators and the ATLAS Group. Goldenberg NA, et al. JAMA. 2022 Jan 11;327(2):129-137. doi: 10.1001/jama.2021.23182. JAMA. 2022. PMID: 35015038 Free PMC article. Clinical Trial.
39 results