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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: wang m. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Multicenter randomized controlled trial on Duration of Therapy for Thrombosis in Children and Young Adults (the Kids-DOTT trial): pilot/feasibility phase findings.
Goldenberg NA, Abshire T, Blatchford PJ, Fenton LZ, Halperin JL, Hiatt WR, Kessler CM, Kittelson JM, Manco-Johnson MJ, Spyropoulos AC, Steg PG, Stence NV, Turpie AG, Schulman S; Kids-DOTT Trial Investigators. Goldenberg NA, et al. J Thromb Haemost. 2015 Sep;13(9):1597-605. doi: 10.1111/jth.13038. Epub 2015 Aug 11. J Thromb Haemost. 2015. PMID: 26118944 Free PMC article. Clinical Trial.
Changes in the amino acid sequence of the recombinant human factor VIIa analog, vatreptacog alfa, are associated with clinical immunogenicity.
Mahlangu JN, Weldingh KN, Lentz SR, Kaicker S, Karim FA, Matsushita T, Recht M, Tomczak W, Windyga J, Ehrenforth S, Knobe K; adept™2 Investigators. Mahlangu JN, et al. J Thromb Haemost. 2015 Nov;13(11):1989-98. doi: 10.1111/jth.13141. Epub 2015 Oct 13. J Thromb Haemost. 2015. PMID: 26362483 Free article. Clinical Trial.
Physical activity in individuals with haemophilia and experience with recombinant factor VIII Fc fusion protein and recombinant factor IX Fc fusion protein for the treatment of active patients: a literature review and case reports.
Wang M, Álvarez-Román MT, Chowdary P, Quon DV, Schafer K. Wang M, et al. Blood Coagul Fibrinolysis. 2016 Oct;27(7):737-744. doi: 10.1097/MBC.0000000000000565. Blood Coagul Fibrinolysis. 2016. PMID: 27116081 Free PMC article. Review.
Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study.
Witkop M, Neff A, Buckner TW, Wang M, Batt K, Kessler CM, Quon D, Boggio L, Recht M, Baumann K, Gut RZ, Cooper DL, Kempton CL. Witkop M, et al. Among authors: wang m. Haemophilia. 2017 Jul;23(4):556-565. doi: 10.1111/hae.13214. Epub 2017 Apr 16. Haemophilia. 2017. PMID: 28419637
Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality of life (P-FiQ) study.
Kempton CL, Wang M, Recht M, Neff A, Shapiro AD, Soni A, Kulkarni R, Buckner TW, Batt K, Iyer NN, Cooper DL. Kempton CL, et al. Among authors: wang m. Patient Prefer Adherence. 2017 Sep 19;11:1603-1612. doi: 10.2147/PPA.S141389. eCollection 2017. Patient Prefer Adherence. 2017. PMID: 29033549 Free PMC article.
Known-group validity of patient-reported outcome instruments and hemophilia joint health score v2.1 in US adults with hemophilia: results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study.
Buckner TW, Wang M, Cooper DL, Iyer NN, Kempton CL. Buckner TW, et al. Among authors: wang m. Patient Prefer Adherence. 2017 Oct 11;11:1745-1753. doi: 10.2147/PPA.S141392. eCollection 2017. Patient Prefer Adherence. 2017. PMID: 29066870 Free PMC article.
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