共 50 条
- [31] A phase 1/2 clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studiesAMERICAN JOURNAL OF HUMAN GENETICS, 2001, 68 (03) : 711 - 722Eng, CMBanikazemi, MGordon, REGoldman, MPhelps, RKim, LGass, AWinston, JDikman, SFallon, JTBrodie, SStacy, CBMehta, DParsons, RNorton, KO'Callaghan, MDesnick, RJ
- [32] Efficacy and safety of ustekinumab in Behcet disease: Results from the prospective phase 2 STELABEC trialJOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY, 2022, 87 (03) : 681 - 684London, JonathanRegent, AlexisDion, JeremieJilet, LeaJachiet, MarieLidove, OlivierCohen-Aubart, FleurAractingi, SelimGuegan, SarahPennaforte, Jean-LoupAbdoul, HendyPuechal, XavierTerrier, Benjamin
- [33] Interim Results from an Ongoing Global Phase 1 Study of Lentiviral Mediated Gene Therapy for Pyruvate Kinase DeficiencyHUMAN GENE THERAPY, 2022, 33 (23-24) : A58 - A58Lopez Lorenzo, J. L.Shah, A. J.Sevilla, J.Navarro, S.Llanos, L.de Camino Gaisse, B. PerezSanchez, S.Zubicaray, J.Glader, B.Chien, M.Bustamante, O.Zeini, M.Choi, G.Nicoletti, E.Rao, G. R.Roncarolo, M. G.Bueren, J. A.Schwartz, J. D.Segovia, J. C.
- [34] UPDATED SAFETY AND EFFICACY RESULTS FROM THE SCISTAR PHASE 1/2A TRIAL OF AST-OPC1 IN PATIENTS WITH SUBACUTE CERVICAL SCIJOURNAL OF NEUROTRAUMA, 2018, 35 (16) : A54 - A55Wirth, Edward, IIIFessler, Richard G.Leslie, DonaldSteinberg, GaryMckenna, SteveLiu, CharlesKurpad, Shekar N.Chen, SusyLebkowski, Jane
- [35] Efficacy and Safety of Durvalumab in Locally Advanced or Metastatic Urothelial Carcinoma Updated Results From a Phase 1/2 Open-label StudyJAMA ONCOLOGY, 2017, 3 (09)Powles, ThomasO'Donnell, Peter H.Massard, ChristopheArkenau, Hendrik-TobiasFriedlander, TerenceW.Hoimes, Christopher J.Lee, Jae LyunOng, MichaelSridhar, Srikala S.Vogelzang, Nicholas J.Fishman, Mayer N.Zhang, JingsongSrinivas, SandyParikh, JigarAntal, JoyceJin, XiaopingGupta, Ashok K.Ben, YongHahn, Noah M.
- [36] ELIGLUSTAT TARTRATE, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1): PHASE 2 RESULTS AFTER 2 YEARSJOURNAL OF INHERITED METABOLIC DISEASE, 2010, 33 : S147 - S147Lukina, E.Peterschmitt, J.Watman, N.Arreguin, E. A.Pastores, G.Lastrebner, M.Dragosky, M.Rosenbaum, H.Phillips, M.Kaper, M.Singh, T.Puga, A. C.
- [37] ELIGLUSTAT, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1): PHASE 2 RESULTS AFTER 3 YEARSJOURNAL OF INHERITED METABOLIC DISEASE, 2011, 34 : S201 - S201Puga, A. C.Lukina, E.Watman, N.Dragosky, M.Iastrebner, M.Pastores, G.Avila Arreguin, E.Phillips, M.Rosenbaum, H.Aguzzi, R.Bertin-Millet, D.Peterschmitt, M. J.
- [38] Safety of Ustekinumab in Inflammatory Bowel Disease: Pooled Safety Analysis of Results from Phase 2/3 StudiesINFLAMMATORY BOWEL DISEASES, 2021, 27 (07) : 994 - 1007Sandborn, William J.Feagan, Brian G.Danese, SilvioO'Brien, Christopher D.Ott, ElyssaMarano, ColleenBaker, ThomasZhou, YiyingVolger, SheriTikhonov, IliaGasink, ChristopherSands, Bruce E.Ghosh, Subrata
- [39] ELIGLUSTAT, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHER DISEASE TYPE 1 (GD1): PHASE 2 RESULTS AFTER 4 YEARSJOURNAL OF INHERITED METABOLIC DISEASE, 2012, 35 : S98 - S98Puga, A. C.Lukina, E.Dragosky, M.Iastrebner, M.Pastores, G.Avila Arreguin, E.Zimran, A.Rosenbaum, H.Watman, N.Sysoeva, E.Aguzzi, R.Ross, L. H.Peterschmitt, M. J.
- [40] A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-DTM Lentiviral Vector for the Treatment of Cerebral AdrenoleukodystrophyMOLECULAR THERAPY, 2016, 24 : S98 - S99Eichler, Florian S.Duncan, ChristineAmartino, HernanAziz-Bose, RazinaBaruchel, AndreDansereau, ColleenDalle, Jean-HuguesGaspar, H. BobbyGissen, PaulKohn, Donald B.Larghero, JeromeLund, Troy C.Miller, Weston P.Musolino, Patricia L.O'Meara, TaraOrchard, Paul J.Paker, Asif M.Shah, Ami J.Shamir, EstherRaymond, Gerald V.Sankar, RamanSmith, Nicholas J. C.Thrasher, Adrian J.Aubourg, PatrickWilliams, David A.