共 27 条
- [1] Achievement of normal factor viii activity following BMN 270 AAV5-FVIII gene transfer: Long-term efficacy and safety from a phase 1/2 study in patients with severe hemophilia AHAEMOPHILIA, 2018, 24 : 29 - 29Pasi, J.Rangarajan, S.Kim, B.Lester, W.Perry, D.Madan, B.Tavakkoli, F.Yang, K.Pierce, G.Wong, W. Y.
- [2] Achievement of normal factor VIII activity following gene transfer with valoctocogene roxaparvovec (BMN 270): Long-term efficacy and safety results in patients with severe hemophilia AHAEMOPHILIA, 2018, 24 : 65 - 65Rangarajan, SavitaKim, BenjaminLester, WillSymington, EmilyMadan, BellaLaffan, MichaelTavakkoli, FatemehPierce, GlennWong, Wing YenPasi, John
- [3] Interim Analysis of Immunogenicity to the Vector Capsid and Transgene - Expressed Human FVIII in a Phase-1/ 2 Clinical Study of Bmn 270, an AAV5-Mediated Gene Therapy for Hemophilia aBLOOD, 2017, 130Long, BrianKim, BenjaminWong, Wing YenYang, KeVettermann, ChristianPryer, NancyHardet, RomainKuranda, KlaudiaVeron, PhilippeMingozzi, FedericoPierce, Glenn F.Schweighardt, Becky
- [4] Interim analysis of immunogenicity to the vector capsid and transgene-expressed human FVIII IN a phase-1/2 clinical study of BMN-270, an AAV5 mediated gene therapy for hemophilia AHAEMOPHILIA, 2018, 24 : 60 - 60Long, B.Kim, B.Wong, W. Y.Yang, K.Vettermann, C.Pryer, N.Hardet, R.Kuranda, K.Veron, P.Mingozzi, F.Pierce, G. F.Schweighardt, B.
- [5] Long-term safety and prophylactic efficacy of once-weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 studyJOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2015, 13 : 6 - 7Shima, M.Hanabusa, H.Taki, M.Matsushita, T.Sato, T.Fukutake, K.Fukazawa, N.Yoneyama, K.Yoshida, H.Takahashi, H.Nogami, K.
- [6] Long-term safety and efficacy of rVIII-SingleChain in patients with severe haemophilia A: Interim results from a phase 3B extension studyHAEMOPHILIA, 2019, 25 : 148 - 149Mahlangu, J.Karim, F. AbdulStasyshyn, O.Korczowski, B.Brainsky, A.Lucas, S.Li, Y.Pabinger, I.
- [7] Results from the Long-Term Follow-Up of Severe Hemophilia B Subjects Previously Enrolled in a Clinical Study of AAV2-FIX Gene Transfer to the LiverMOLECULAR THERAPY, 2012, 20 : S28 - S29Wellman, Jennifer A.Mingozzi, FedericoOzelo, Margareth C.Arruda, ValderPodsakoff, GregChen, YifengKonkle, Barbara A.Blatt, Philip M.Hoots, KeithRaffini, Leslie J.Rasko, JohnRagni, Margaret V.High, Katherine A.
- [8] Long-term efficacy and safety of tildrakizumab in Japanese patients with moderate to severe plaque psoriasis: Results from a 5-year extension of a phase 3 study (reSURFACE 1)JOURNAL OF DERMATOLOGY, 2021, 48 (06): : 844 - 852Imafuku, ShinichiNakagawa, HidemiIgarashi, AtsuyukiMorita, AkimichiOkubo, YukariSano, ShigetoshiTada, YayoiNemoto, OsamuRozzo, Stephen J.Kawamura, MasakiOhtsuki, Mamitaro
- [9] Long-term safety and efficacy of lentiglobin gene therapy in patients with transfusion-dependent β-thalassemia following completion of the phase 1/2 northstar studyBONE MARROW TRANSPLANTATION, 2019, 54 : 352 - 354Hongeng, SuradejThompson, AlexisKwiatkowski, JanetRasko, JohnSchiller, GaryVichinsky, ElliottKletzel, MorrisHo, P. JoyAnurathapan, UsanaratSchmidt, ManfredCavazzana, MarinaLeboulch, PhilippeDeary, BrianaChen, YingAsmal, MohammedWalters, Mark
- [10] SAFETY AND EFFICACY OF THE ANTI-TISSUE FACTOR PATHWAY INHIBITOR MARSTACIMAB IN PARTICIPANTS WITH SEVERE HEMOPHILIA WITHOUT INHIBITORS: RESULTS FROM THE PHASE 3 BASIS TRIAL AND ONGOING LONG-TERM EXTENSION STUDYAMERICAN JOURNAL OF HEMATOLOGY, 2024, 100 : 4 - 4Acharya, Suchitra S.Matino, DavidePalladino, AndrewHwang, EunheeMcDonald, ReginaTaylor, Carrie TurichTeeter, John