Kwiatkowski, Janet L.

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 684

Controlled Trial of Transfusions for Silent Cerebral Infarcts in Sickle Cell Anemia

DeBaun, M. R., Gordon, M., McKinstry, R. C., Noetzel, M. J., White, D. A., Sarnaik, S. A., Meier, E. R., Howard, T. H., Majumdar, S., Inusa, B. P. D., et al. (2014) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 545

Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial

Ware, R. E., Davis, B. R., Schultz, W. H., Brown, R. C., Aygun, B., Sarnaik, S., Odame, I., Fuh, B., George, A., Owen, W., et al. (2016) · The Lancet

Hemoglobinopathies and Related DisordersNeonatal Health and Biochemistry

Cited by 515

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Locatelli, F., Lang, P., Wall, D., Meisel, R., Corbacioglu, S., Li, A. M., de la Fuente, J., Shah, A. J., Carpenter, B., Kwiatkowski, J. L., et al. (2024) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 253

Inflammation and oxidant-stress in -thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial

Walter, P. B., Macklin, E. A., Porter, J., Evans, P., Kwiatkowski, J. L., Neufeld, E. J., Coates, T., Giardina, P. J., Vichinsky, E., Olivieri, N., et al. (2008) · Haematologica

Hemoglobinopathies and Related DisordersIron Metabolism and Disorders

Cited by 87

Gene therapy of hemoglobinopathies: progress and future challenges

Ikawa, Y., Miccio, A., Magrin, E., Kwiatkowski, J. L., Rivella, S., & Cavazzana, M. (2019) · Human Molecular Genetics

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 86

Effects of hydroxyurea treatment for patients with hemoglobin SC disease

Luchtman‐Jones, L., Pressel, S., Hilliard, L., Brown, R. C., Smith, M. G., Thompson, A. A., Lee, M. T., Rothman, J., Rogers, Z. R., Owen, W., et al. (2016) · American Journal of Hematology

Hemoglobinopathies and Related DisordersHemophilia Treatment and Research

Cited by 86

Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thalassemia Longitudinal Cohort

Tubman, V. N., Fung, E. B., Vogiatzi, M., Thompson, A. A., Rogers, Z. R., Neufeld, E. J., & Kwiatkowski, J. L. (2015) · Journal of Pediatric Hematology/Oncology

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 68

Effect of transfusion therapy on transcranial doppler ultrasonography velocities in children with sickle cell disease

Kwiatkowski, J. L., Yim, E., Miller, S., Adams, R. J., & for the STOP 2 Study Investigators (2011) · Pediatric Blood & Cancer

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 49

Intracranial aneurysms in sickle cell anemia: clinical and imaging findings

Nabavizadeh, S. A., Vossough, A., Ichord, R. N., Kwiatkowski, J., Pukenas, B. A., Smith, M. J., Storm, P. B., Zager, E. L., & Hurst, R. W. (2016) · Journal of NeuroInterventional Surgery

Intracranial Aneurysms: Treatment and ComplicationsHemoglobinopathies and Related Disorders

Cited by 43

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Kanter, J., Tisdale, J. F., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Huang, W., Ribeil, J.-A., et al. (2019) · Blood

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 21

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Kanter, J., Thompson, A. A., Kwiatkowski, J. L., Parikh, S., Mapara, M., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Gupta, A. O., Zhang, L., et al. (2023) · Blood

Hemoglobinopathies and Related DisordersNeurogenetic and Muscular Disorders Research

Cited by 15

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Thompson, A. A., Olson, T. S., Walters, M. C., Porter, J. B., Schneiderman, J., Hongeng, S., Kulozik, A., Cavazzana, M., Sauer, M. G., Thrasher, A. J., et al. (2023) · Blood

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 6