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The translational gap for gene therapies in low- and middle-income countries.
Doxzen KW, Adair JE, Fonseca Bazzo YM, Bukini D, Cornetta K, Dalal V, Guerino-Cunha RL, Hongeng S, Jotwani G, Kityo-Mutuluuza C, Lakshmanan K, Mahlangu J, Makani J, Mathews V, Ozelo MC, Rangarajan S, Scholefield J, Batista Silva Júnior J, McCune JM. Doxzen KW, et al. Among authors: mahlangu j. Sci Transl Med. 2024 May 8;16(746):eadn1902. doi: 10.1126/scitranslmed.adn1902. Epub 2024 May 8. Sci Transl Med. 2024. PMID: 38718130 Review.
Key issues in inhibitor management in patients with haemophilia.
Gomez K, Klamroth R, Mahlangu J, Mancuso ME, Mingot ME, Ozelo MC. Gomez K, et al. Among authors: mahlangu j. Blood Transfus. 2014 Jan;12 Suppl 1(Suppl 1):s319-29. doi: 10.2450/2013.0246-12. Epub 2013 Dec 3. Blood Transfus. 2014. PMID: 24333092 Free PMC article. Review. No abstract available.
The potential correlation between patient-reported symptoms and the use of additional haemostatic medication for joint bleeding in haemophilia patients with inhibitors: a post hoc exploratory analysis of recombinant activated factor VII data from the ADEPT2 trial.
Lentz SR, Rangarajan S, Karim FA, Andersen PD, Arkhammar P, Rosu G, Mahlangu J. Lentz SR, et al. Among authors: mahlangu j. Blood Coagul Fibrinolysis. 2017 Apr;28(3):224-229. doi: 10.1097/MBC.0000000000000584. Blood Coagul Fibrinolysis. 2017. PMID: 27427786 Free PMC article. Clinical Trial.
Real-World Rates of Bleeding, Factor VIII Use, and Quality of Life in Individuals with Severe Haemophilia A Receiving Prophylaxis in a Prospective, Noninterventional Study.
Kenet G, Chen YC, Lowe G, Percy C, Tran H, von Drygalski A, Trossaërt M, Reding M, Oldenburg J, Mingot-Castellano ME, Park YS, Peyvandi F, Ozelo MC, Mahlangu J, Quinn J, Huang M, Reddy DB, Kim B. Kenet G, et al. Among authors: mahlangu j. J Clin Med. 2021 Dec 18;10(24):5959. doi: 10.3390/jcm10245959. J Clin Med. 2021. PMID: 34945255 Free PMC article.
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. Among authors: mahlangu j. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
Haemophilia gene therapy-Update on new country initiatives.
Reiss UM, Mahlangu J, Ohmori T, Ozelo MC, Srivastava A, Zhang L. Reiss UM, et al. Among authors: mahlangu j. Haemophilia. 2022 May;28 Suppl 4:61-67. doi: 10.1111/hae.14512. Haemophilia. 2022. PMID: 35521726 Review.
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B.
Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Shapiro AD, et al. Among authors: mahlangu j. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230. Blood Adv. 2023. PMID: 36848635 Free PMC article.
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.
Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Mahlangu J, et al. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13. Blood. 2014. PMID: 24227821 Free PMC article. Clinical Trial.
178 results