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- [11] Efficacy and safety of sigvotatug vedotin, an investigational ADC, in NSCLC: Updated phase 1 results (SGNB6A-001)JOURNAL OF CLINICAL ONCOLOGY, 2024, 42 (16)Peters, SolangeHollebecque, AntoineSehgal, KartikLopez, Juanita SuzanneCalvo, EmilianoDowlati, AfshinBockorny, BrunoPerez, Cesar AugustoSanborn, Rachel E.Patnaik, AmitaFontana, ElisaGalvao, VladimirKingsley, EdPatilea-Vrana, GabrielaWang, TianhuaKnowles, ScottPiha-Paul, Sarina A.
- [12] Eliglustat Tartrate, a Novel Investigational Oral Substrate Reduction Therapy for Gaucher Disease Type 1: Updated Phase 2 ResultsBLOOD, 2010, 116 (21) : 1551 - 1551Lukina, ElenaPeterschmitt, JudithWatman, NoraDragosky, MartaLastrebner, MarceloPastores, GregoryArreguin, Elsa AvilaRosenbaum, HannaPhillips, MiciKaper, MathildeHaque, ArshadulPuga, Ana Cristina
- [13] SPK-8011: Preliminary results from a phase 1/2 trial of investigational gene therapy for hemophilia AHAEMOPHILIA, 2018, 24 : 30 - 31George, L. A.Ragni, M.Sullivan, S. K.Barrett, J. C.Samelson-Jones, B. J.Cuker, A.Cole, G.Wright, F.Chen, Y.Hui, D. J.Liu, Y.Wachtel, K.Takefman, D.Couto, L. B.Reape, K.Carr, M. E.Anguela, X. M.High, K. A.
- [14] Pharmacokinetics, safety, and efficacy of daridorexant in Japanese subjects: Results from phase 1 and 2 studiesJOURNAL OF SLEEP RESEARCH, 2025, 34 (01)Uchiyama, MakotoMishima, KazuoYagi, TomokoYoshihara, TatsuyaEto, TakashiMuehlan, ClemensTogo, OsamuInoue, Yuichi
- [15] Efficacy and safety of LentiGlobin gene therapy in patients with transfusion-dependent β-thalassaemia and non-β0/β0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar-2 studiesBRITISH JOURNAL OF HAEMATOLOGY, 2019, 185 : 23 - 23Porter, J. B.Walters, M. C.Kwiatkowski, J. L.Hongeng, S.Rasko, J. E. J.Sauer, M. G.Thrasher, A. J.Thuret, I.Schiller, G. J.Elliot, H.Deary, B.Chen, Y.Tao, G.Asmal, M.Locatelli, F.Thompson, A. A.
- [16] EFFICACY AND SAFETY OF LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA AND NON-β0/β0 GENOTYPES: UPDATED RESULTS FROM THE COMPLETED PHASE 1/2 NORTHSTAR AND ONGOING PHASE 3 NORTHSTAR-2 STUDIESCYTOTHERAPY, 2019, 21 (05) : S14 - S14Rasko, J.Walters, M.Kwiatkowski, J.Hongeng, S.Porter, J.Sauer, M.Thrasher, A.Thuret, I.Schiller, G.Elliot, H.Deary, B.Chen, Y.Tao, G.Asmal, M.Locatelli, F.Thompson, A.
- [17] Efficacy and safety of migalastat, an oral pharmacologic chaperone for Fabry disease: results from two randomized phase 3 studies, FACETS and ATTRACTMOLECULAR GENETICS AND METABOLISM, 2017, 120 (1-2) : S45 - S46Feldt-Rasmussen, UllaGiugliani, RobertoGermain, Dominique P.Hughes, Derralynn A.Wilcox, William R.Schiffmann, RaphaelBichet, Daniel G.Jovanovic, AnaBratkovic, DragoCastelli, Jeffrey P.Yu, JulieSkuban, NinaBarth, Jay A.
- [18] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric PatientsMOLECULAR THERAPY, 2021, 29 (04) : 42 - 43Lopez Lorenzo, Jose LuisShah, Ami J.Navarro, SusanaSevilla, JulianLlanos, LuciaPerez Camino de Gaisse, BegonaSanchez, SolGlader, BertilChien, MayQuintana-Bustamante, OscarBeard, Brian C.Law, Kenneth M.Zeini, MiriamChoi, GraceNicoletti, EileenRao, Gayatri R.Roncarolo, Maria GraziaBueren, Juan A.Schwartz, Jonathan D.Segovia, Jose C.
- [19] Hematopoietic Stem Cell Gene Therapy for Cystinosis: Updated Results from a Phase 1/2 Clinical TrialMOLECULAR THERAPY, 2022, 30 (04) : 29 - 29Cherqui, Stephanie
- [20] Results from a phase I safety trial of hAADC gene therapy for Parkinson diseaseNEUROLOGY, 2008, 70 (21) : 1980 - 1983Eberling, J. L.Jagust, W. J.Christine, C. W.Starr, P.Larson, P.Bankiewicz, K. S.Aminoff, M. J.