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Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-134940

Published: 2020

Journal Articles

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Julie Kanter, MD, John F. Tisdale, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Ren Chen, Meghan E. Gallagher, MSc, Yuchen Ding, PhD, Sunita Goyal, MD, Clark Paramore, MPH, Alexis A. Thompson, MD, Mark C. Walters, MD

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-136193

Published: 2020

Journal Articles

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Janet L. Kwiatkowski, MD MSCE, Mark C. Walters, MD, Suradej Hongeng, MD, Franco Locatelli, MD PhD, John E.J. Rasko, BSc, MBBS, PhD, Marina Cavazzana, MD PhD, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 51–52.

https://doi.org/10.1182/blood-2020-135850

Published: 2020

Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 949.

https://doi.org/10.1182/blood-2019-125352

Published: 2019

Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Blood (2019) 134 (Supplement_1): 2175.

https://doi.org/10.1182/blood-2019-123069

Published: 2019

Journal Articles

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

Kathryn Addonizio, BA, Hanny Al-Samkari, MD, Bertil Glader, MD PhD, D Holmes Morton, MD, Satheesh Chonat, MD, Alexis A. Thompson, MD MPH, Kevin H. M. Kuo, MDMSc,FRCPC, Yaddanapudi Ravindranath, MBBS, Heng Wang, MD PhD, Jennifer A. Rothman, MD, Janet L. Kwiatkowski, MD MSCE, Charles Kung, PhD, Penelope A. Kosinski, MS, Hasan Al-Sayegh, MS, Wendy B. London, PhD, Rachael F. Grace, MD

Blood (2019) 134 (Supplement_1): 3515.

https://doi.org/10.1182/blood-2019-125248

Published: 2019

Journal Articles

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Rachael F. Grace, MD, D. Mark Layton, MB BS, FRCP, FRCPCH, Frédéric Galactéros, MD PhD, Wilma Barcellini, MD, Eduard J. van Beers, MD PhD, Hassan M. Yaish, MD, Yaddanapudi Ravindranath, MBBS, Kevin H. M. Kuo, MDMSc,FRCPC, Sujit Sheth, MD, Janet L. Kwiatkowski, MD MSCE, Lei Hua, PhD, Peter F. Hawkins, PhD, Chris Mix, MD, Bertil Glader, MD PhD

Blood (2019) 134 (Supplement_1): 3512.

https://doi.org/10.1182/blood-2019-123406

Published: 2019

Journal Articles

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Alexis A. Thompson, MD MPH, Mark C. Walters, MD, Janet L. Kwiatkowski, MD MSCE, Suradej Hongeng, MD, John B. Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Heidi Elliot, Ge Tao, PhD, Richard A. Colvin, MD PhD, Franco Locatelli, MD PhD

Blood (2019) 134 (Supplement_1): 3543.

https://doi.org/10.1182/blood-2019-126046

Published: 2019

Journal Articles

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Angela R. Smith, MD MS, Gary J. Schiller, MD, Gregory M Vercellotti, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Erica B. Esrick, MD, David A. Williams, MD, Weston P. Miller, MD, Adrian Woolfson, MD PhD, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 3544.

https://doi.org/10.1182/blood-2019-125743

Published: 2019

Journal Articles

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

Blood (2019) 134 (Supplement_1): 618.

https://doi.org/10.1182/blood-2019-122062

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

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

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

Janet L. Kwiatkowski, MD MSCE, Julie Kanter, MD, Heather J. Fullerton, MD, Jenifer Voeks, Ellen Debenham, RN, David G Brown, Lynette J Brown, Robert J. Adams, MD MS

Blood (2015) 126 (23): 68.

https://doi.org/10.1182/blood.V126.23.68.68

Published: 2015

Journal Articles

Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)

Alexis A. Thompson, MD, John E Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD MSCE, Gary Schiller, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Mark C. Walters, MD

Blood (2014) 124 (21): 549.

https://doi.org/10.1182/blood.V124.21.549.549

Published: 2014

Journal Articles

Transcranial Doppler Ultrasound (TCD) Outcomes for Children with Prior Normal TCD: Post-STOP Study Results

Janet L. Kwiatkowski, MD MSCE, Heather Fullerton, MD MAS, Jennifer Voeks, Lynette Brown, Ellen Debenham, RN, Robert J. Adams, MD MS

Blood (2014) 124 (21): 562.

https://doi.org/10.1182/blood.V124.21.562.562

Published: 2014

NARROW

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Long-Term Efficacy and Safety of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia: Results in Patients with up to 6 Years of Follow-up

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency

Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency

Long-Term Safety and Efficacy of Mitapivat (AG-348), a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency: The DRIVE PK Study

Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Non-β0/β0 Genotypes

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

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

Ischemic Stroke in Children and Young Adults with Sickle Cell Disease (SCD) in the Post-STOP Era

Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)

Transcranial Doppler Ultrasound (TCD) Outcomes for Children with Prior Normal TCD: Post-STOP Study Results

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American Society of Hematology

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ASH Publications

American Society of Hematology

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