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Journal Articles

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

Blood (2024) 144 (Supplement 1): 7454.

https://doi.org/10.1182/blood-2024-204115

Published: 2024

Journal Articles

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Blood (2024) 144 (Supplement 1): 3583.1.

https://doi.org/10.1182/blood-2024-201734

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2024) 144 (Supplement 1): 512.

https://doi.org/10.1182/blood-2024-205779

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2024) 144 (Supplement 1): 4954.

https://doi.org/10.1182/blood-2024-204001

Published: 2024

Journal Articles

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Danielle E. Arnold, Suhag Parikh, Brent Logan, Rebecca A. Marsh, Linda M. Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J. Si Lim, Eyal Grunebaum, E. Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K. Prasad, Jennifer R. Heimall, Fabien Touzot, Lauri M. Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R. Oshrine, Jeffrey J. Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D. E. Cuvelier, Lisa R. Forbes Satter, Caridad Martinez, Mark T. Vander Lugt, Lolie C. Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E. Prockop, Blachy J. Dávila Saldaña, Victor Aquino, Larisa A. Broglie, Christen L. Ebens, Lisa M. Madden, Kenneth DeSantes, Jordan Milner, Hemalatha G. Rangarajan, Ami J. Shah, Alfred P. Gillio, Alan P. Knutsen, Holly K. Miller, Theodore B. Moore, Pamela Graham, Andrea Bauchat, Nancy J. Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J. Dorsey, Olga Birbrayer, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson, Harry L. Malech, Elizabeth M. Kang

Blood (2023) 142 (24): 2105–2118.

https://doi.org/10.1182/blood.2022019586

Published: 2023

Includes: Supplemental data

Journal Articles

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2023) 142 (Supplement 1): 1053.

https://doi.org/10.1182/blood-2023-190534

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2023-190139

Published: 2023

Journal Articles

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Ami J. Shah, José Luis López Lorenzo, Julián Sevilla, Susana Navarro, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, Bert Glader, May Chien, Oscar Quintana Bustamante, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R. Rao, Maria Grazia Roncarolo, Juan A. Bueren, Jonathan D. Schwartz, José C. Segovia

Blood (2022) 140 (Supplement 1): 4902–4903.

https://doi.org/10.1182/blood-2022-170948

Published: 2022

Journal Articles

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Blood (2022) 140 (Supplement 1): 4899–4901.

https://doi.org/10.1182/blood-2022-166881

Published: 2022

Journal Articles

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier, Brent R. Logan, Susan E. Prockop, Rebecca H. Buckley, Caroline Y. Kuo, Linda M. Griffith, Xuerong Liu, Alison Yip, Michael S. Hershfield, Paul G. Ayoub, Theodore B. Moore, Morna J. Dorsey, Richard J. O’Reilly, Neena Kapoor, Sung-Yun Pai, Malika Kapadia, Christen L. Ebens, Lisa R. Forbes Satter, Lauri M. Burroughs, Aleksandra Petrovic, Deepak Chellapandian, Jennifer Heimall, David C. Shyr, Ahmad Rayes, Jeffrey J. Bednarski, Sharat Chandra, Shanmuganathan Chandrakasan, Alfred P. Gillio, Lisa Madden, Troy C. Quigg, Emi H. Caywood, Blachy J. Dávila Saldaña, Kenneth DeSantes, Hesham Eissa, Frederick D. Goldman, Jacob Rozmus, Ami J. Shah, Mark T. Vander Lugt, Monica S. Thakar, Roberta E. Parrott, Caridad Martinez, Jennifer W. Leiding, Troy R. Torgerson, Michael A. Pulsipher, Luigi D. Notarangelo, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Jennifer M. Puck, Donald B. Kohn

Blood (2022) 140 (7): 685–705.

https://doi.org/10.1182/blood.2022016196

Published: 2022

Includes: Supplemental data

Journal Articles

Functional Immune Tolerance Induced By Sequential Hematopoietic Stem Cell-Solid Organ Transplantation

Alice Bertaina, Giulia Barbarito, Vasavi V. Ramachandran, Karen Kristovich, Elizabeth Amanda Lippner, Sahar Fathallah-Shaykh, Amira Al-Uzri, Ami J Shah, Geraldine Aubert, Priscila Ferreira Slepicka, Linda Oppizzi, Rajni Agarwal, Maria Grazia Roncarolo, Amy Gallo, Waldo Concepcion, Kenneth I. Weinberg, Robertson Parkman, David B Lewis, Paul C Grimm

Blood (2021) 138 (Supplement 1): 1818.

https://doi.org/10.1182/blood-2021-149920

Published: 2021

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Blood (2021) 138 (Supplement 1): 563.

https://doi.org/10.1182/blood-2021-148161

Published: 2021

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Blood (2020) 136 (Supplement 1): 47.

https://doi.org/10.1182/blood-2020-137246

Published: 2020

Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

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

https://doi.org/10.1182/blood-2020-137762

Published: 2020

Journal Articles

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Lauri M. Burroughs, Aleksandra Petrovic, Ruta Brazauskas, Xuerong Liu, Linda M. Griffith, Hans D. Ochs, Jack J. Bleesing, Stephanie Edwards, Christopher C. Dvorak, Sonali Chaudhury, Susan E. Prockop, Ralph Quinones, Frederick D. Goldman, Troy C. Quigg, Shanmuganathan Chandrakasan, Angela R. Smith, Suhag Parikh, Blachy J. Dávila Saldaña, Monica S. Thakar, Rachel Phelan, Shalini Shenoy, Lisa R. Forbes, Caridad Martinez, Deepak Chellapandian, Evan Shereck, Holly K. Miller, Neena Kapoor, Jessie L. Barnum, Hey Chong, David C. Shyr, Karin Chen, Rolla Abu-Arja, Ami J. Shah, Katja G. Weinacht, Theodore B. Moore, Avni Joshi, Kenneth B. DeSantes, Alfred P. Gillio, Geoffrey D. E. Cuvelier, Michael D. Keller, Jacob Rozmus, Troy Torgerson, Michael A. Pulsipher, Elie Haddad, Kathleen E. Sullivan, Brent R. Logan, Donald B. Kohn, Jennifer M. Puck, Luigi D. Notarangelo, Sung-Yun Pai, David J. Rawlings, Morton J. Cowan

Blood (2020) 135 (23): 2094–2105.

https://doi.org/10.1182/blood.2019002939

Published: 2020

Journal Articles

Improved Outcomes After Reduced Intensity Conditioning Matched Unrelated Donor Hematopoietic Stem Cell Transplantation In Children With Chronic Granulomatous Disease

Bhakti Mehta, MD, Kris M. Mahadeo, MD, Ami J Shah, MD, Neena Kapoor, MD, Hisham Abdel-Azim, MD

Blood (2013) 122 (21): 2272.

https://doi.org/10.1182/blood.V122.21.2272.2272

Published: 2013

Journal Articles

Older Age, Use Of Myeloablative Regimens For Malignant Diseases and Chronic Graft-Versus-Host Disease Are Risk Factors For Avascular Necrosis Of Bone After Allogeneic Hematopoietic Cell Transplantation In Children and Adolescents

Xianxin Li, MD, Ruta Brazauskas, PhD, Zhiwei Wang, MS, Amal Al-Seraihy, K. Scott Baker, MD, MS, Jean-Yves Cahn, MD, Haydar A. Frangoul, MD, James L. Gajewski, MD, Gregory A. Hale, MD, Jack W. Hsu, MD, Rammurti T. Kamble, MD, Hillard M. Lazarus, MD, Richard T. Maziarz, MD, Bipin N. Savani, MD, Ami J. Shah, MD, Mohamed L. Sorror, MD, MSc, William A. Wood, MD, MPH, Navneet S. Majhail, MD, MS

Blood (2013) 122 (21): 917.

https://doi.org/10.1182/blood.V122.21.917.917

Published: 2013

Journal Articles

Long Term Humoral Immune Reconstitution Kinetics After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Children

Amro Elshoury, MD, Neena Kapoor, MD, Ami J Shah, MD, Bhakti Mehta, MD, Kris M. Mahadeo, MD, Hisham Abdel-Azim, MD

Blood (2013) 122 (21): 4627.

https://doi.org/10.1182/blood.V122.21.4627.4627

Published: 2013

Journal Articles

Impact of Thymic Function On Chronic Graft-Versus-Host Disease (cGVHD).

Kris M. Mahadeo, M.D., M.P.H., Ami J Shah, MD, Bernadette Masinsin, BS, Neena Kapoor, MD, Hisham Abdel-Azim, MD, Robertson Parkman, M.D.

Blood (2012) 120 (21): 3052.

https://doi.org/10.1182/blood.V120.21.3052.3052

Published: 2012

Journal Articles

Chronic Graft-Versus-Host Disease and Systemic Sclerosis: The Same Pathophysiology?,

Robertson Parkman, MD, Ami J Shah, MD, Bernadette Masinsin, BS, Neena Kapoor, MD, Hisham Abdel-Azim, MD, Andreas Reiff, MD

Blood (2011) 118 (21): 4076.

https://doi.org/10.1182/blood.V118.21.4076.4076

Published: 2011

NARROW

Health-Related Quality-of-Life Improvements after Exagamglogene Autotemcel in Patients with Transfusion-Dependent Beta-Thalassemia

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Functional Immune Tolerance Induced By Sequential Hematopoietic Stem Cell-Solid Organ Transplantation

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

Improved Outcomes After Reduced Intensity Conditioning Matched Unrelated Donor Hematopoietic Stem Cell Transplantation In Children With Chronic Granulomatous Disease

Older Age, Use Of Myeloablative Regimens For Malignant Diseases and Chronic Graft-Versus-Host Disease Are Risk Factors For Avascular Necrosis Of Bone After Allogeneic Hematopoietic Cell Transplantation In Children and Adolescents

Long Term Humoral Immune Reconstitution Kinetics After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Children

Impact of Thymic Function On Chronic Graft-Versus-Host Disease (cGVHD).

Chronic Graft-Versus-Host Disease and Systemic Sclerosis: The Same Pathophysiology?,

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American Society of Hematology

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American Society of Hematology

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