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Journal Articles

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Lorenzo Tozzi, Giulia Schiroli, Yaser Heshmati, Yu Cao, Michael T. Monte, Apiwat Wangweerawong, James B. Rottman, Rahul Palchaudhuri, Jean-Antoine Ribeil, John Manis, Hari Pujar, William Querbes, Anne Bothmer, Cecilia Cotta-Ramusino, Z. Jane Wang, William E. Salomon, Michael C Holmes

Blood (2024) 144 (Supplement 1): 515.

https://doi.org/10.1182/blood-2024-208626

Published: 2024

Journal Articles

Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease

Julia Z Xu, Enrico M Novelli, Jean-Antoine Ribeil, Andreas Glenthøj, Srila Gopal, Hanny Al-Samkari, Modupe Idowu, Jenny Despotovic, Spurthi Patil, Xiaoshu Dai, Abdullah Al Masud, Michael Callaghan, Fuad El Rassi

Blood (2024) 144 (Supplement 1): 2496.

https://doi.org/10.1182/blood-2024-203509

Published: 2024

Journal Articles

SNAP: Implementation of Supportive Non-Invasive Ventilation for Acute Chest Syndrome Prevention in Adults with Sickle Cell Disease

Galia Pollock, Anish Vora, Lillian McMahon, Jean-Antoine Ribeil, Amy E. Sobota, Charlene Sylvestre, Robyn T. Cohen, Elizabeth S. Klings

Blood (2023) 142 (Supplement 1): 2522.

https://doi.org/10.1182/blood-2023-182206

Published: 2023

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

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Blood (2019) 134 (Supplement_1): 3358.

https://doi.org/10.1182/blood-2019-127393

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

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Nicolas Hebert, Elisa Magrin, PhD, Annarita Miccio, PhD, Kiger Laurent, PhD, Nguyen-Peyre Kim-Anh, PhD, Laure Joseph, MD, Alessandra Magnani, MD PhD, Chloé Couzin, MD, Wassim El Nemer, PhD, France Pirenne, MD PhD, Olivier Negre, PhD, Jean-Antoine Ribeil, MD PhD, Isabelle André-Schmutz, PhD, Pablo Bartolucci, MD PhD, Marina Cavazzana, MD PhD

Blood (2018) 132 (Supplement 1): 2195.

https://doi.org/10.1182/blood-2018-99-115973

Published: 2018

Journal Articles

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

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

Blood (2018) 132 (Supplement 1): 1026.

https://doi.org/10.1182/blood-2018-99-113480

Published: 2018

Journal Articles

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

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

Blood (2018) 132 (Supplement 1): 1080.

https://doi.org/10.1182/blood-2018-99-113477

Published: 2018

Journal Articles

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Blood (2018) 132 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2018-99-111770

Published: 2018

Journal Articles

Management of Infections in SCD Patients Treated with HU in the Framework of Escort HU

Frédéric Galactéros, MDPhD, Malika Benkerrou, MD, Mariane De Montalembert, MD PhD, Jean-Antoine Ribeil, MD, Corinne Pondarré, MD PhD, Jean Benoît Arlet, MD, Uwe Kordes, MD MRCP, Regine Grosse, MD, Valentine Brousse, MD, Lena Oevermann, MD, Stephan Lobitz, MD

Blood (2017) 130 (Supplement 1): 3543.

https://doi.org/10.1182/blood.V130.Suppl_1.3543.3543

Published: 2017

Journal Articles

Management of Renal Dysfunctions in Escort-HU Study

Pablo Bartolucci, MD PhD, Gylna Loko, MD, Corinne Charneau, MD, Jean-Antoine Ribeil, MD, Nathalie Lemonne, MD, Stephan Lobitz, MD, Malika Benkerrou, MD, Frederic Galacteros, MDPhD

Blood (2017) 130 (Supplement 1): 3528.

https://doi.org/10.1182/blood.V130.Suppl_1.3528.3528

Published: 2017

Journal Articles

Management of Male Fertility in Sickle Cell Disease before and after Hydroxycarbamide

Anoosha Habibi, MD, Elena Foïs, MD, Giovanna Cannas, MD, Jean-Antoine Ribeil, MD, Pierre Cougoul, MD, Emmanuelle Bernit, MD, Pablo Bartolucci, MD PhD, Frederic Galacteros, MDPhD

Blood (2017) 130 (Supplement 1): 2260.

https://doi.org/10.1182/blood.V130.Suppl_1.2260.2260

Published: 2017

Journal Articles

Prospective Controlled Trial on Endurance Exercise Training in Adult Sickle Cell Disease Patients

Barnabas Gellen, Laurent Messonnier, Angele Merlet, Etienne Audureau, Thomas Rupp, Sandrine Peyrot, Cyril Martin, PhD, Jean-Antoine Ribeil, MD PhD, jean Benoit Arlet, Frédéric Galactéros, MDPhD, Leonard Feasson, Pablo Bartolucci, MD PhD

Blood (2017) 130 (Supplement 1): 991.

https://doi.org/10.1182/blood.V130.Suppl_1.991.991

Published: 2017

Journal Articles

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Blood (2017) 130 (Supplement 1): 4609.

https://doi.org/10.1182/blood.V130.Suppl_1.4609.4609

Published: 2017

Journal Articles

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Louis MINE, Thao Nguyen-Khoa, Birch Allaf, Jean-Antoine Ribeil, MD PhD, Christelle Remus, Valérie Gauthereau, Sarah Enouz, Jason S. KIM, Xiaoxi Yang, Eliane Gluckman, MD PhD, Arnold Munnich, Marina Cavazzana, MD PhD, Robert Girot, MD

Blood (2016) 128 (22): 1308.

https://doi.org/10.1182/blood.V128.22.1308.1308

Published: 2016

Journal Articles

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Laure Joseph, MD, Anoosha Habibi, MD, Giovanna Cannas, MD, Stephanie Ngo, MD, Sylvain Le Jeune, MD, Ersi Voskaridou, MD, Elena Foïs, MD, Caroline Makowski, MD, Nathalie Lemonne, MD, Narcisse Elenga, MD, Yves Ville, MD PhD, Alexandra Benachi, MDPhD, Robert Girot, MD, Marina Cavazzana, MD PhD, Jean-Antoine Ribeil, MD PhD

Blood (2016) 128 (22): 1309.

https://doi.org/10.1182/blood.V128.22.1309.1309

Published: 2016

Journal Articles

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Blood (2016) 128 (22): 4856.

https://doi.org/10.1182/blood.V128.22.4856.4856

Published: 2016

Journal Articles

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Blood (2016) 128 (22): 2311.

https://doi.org/10.1182/blood.V128.22.2311.2311

Published: 2016

Journal Articles

Comparison of the Safety Profile of Sickle Cell Disease Patients Treated with Hydroxycarbamide in Off-Label Versus in-Label Prescriptions in the Escort-HU Non Interventional, Prospective, Observational Open-Label Cohort Study

Ersi Voskaridou, MD, Stephan Lobitz, MD, Uwe Kordes, MD MRCP, Regine Grosse, MD, Valentine Brousse, MD, Malika Benkerrou, MD, Mariane De Montalembert, MD PhD, Jean-Antoine Ribeil, MD PhD, Frederic Galacteros, MD PhD

Blood (2016) 128 (22): 2497.

https://doi.org/10.1182/blood.V128.22.2497.2497

Published: 2016

NARROW

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease

SNAP: Implementation of Supportive Non-Invasive Ventilation for Acute Chest Syndrome Prevention in Adults with Sickle Cell Disease

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

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

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

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Management of Infections in SCD Patients Treated with HU in the Framework of Escort HU

Management of Renal Dysfunctions in Escort-HU Study

Management of Male Fertility in Sickle Cell Disease before and after Hydroxycarbamide

Prospective Controlled Trial on Endurance Exercise Training in Adult Sickle Cell Disease Patients

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Comparison of the Safety Profile of Sickle Cell Disease Patients Treated with Hydroxycarbamide in Off-Label Versus in-Label Prescriptions in the Escort-HU Non Interventional, Prospective, Observational Open-Label Cohort Study

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American Society of Hematology

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ASH Publications

American Society of Hematology

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