Search | VHL CLAP/WR-PAHO/WHO

1.

Exagamglogene Autotemcel for Severe Sickle Cell Disease.

Frangoul, Haydar; Locatelli, Franco; Sharma, Akshay; Bhatia, Monica; Mapara, Markus; Molinari, Lyndsay; Wall, Donna; Liem, Robert I; Telfer, Paul; Shah, Ami J; Cavazzana, Marina; Corbacioglu, Selim; Rondelli, Damiano; Meisel, Roland; Dedeken, Laurence; Lobitz, Stephan; de Montalembert, Mariane; Steinberg, Martin H; Walters, Mark C; Eckrich, Michael J; Imren, Suzan; Bower, Laura; Simard, Christopher; Zhou, Weiyu; Xuan, Fengjuan; Morrow, Phuong Khanh; Hobbs, William E; Grupp, Stephan A.

N Engl J Med ; 390(18): 1649-1662, 2024 May 09.

Article in English | MEDLINE | ID: mdl-38661449

2.

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.

Kanter, Julie; Walters, Mark C; Krishnamurti, Lakshmanan; Mapara, Markus Y; Kwiatkowski, Janet L; Rifkin-Zenenberg, Stacey; Aygun, Banu; Kasow, Kimberly A; Pierciey, Francis J; Bonner, Melissa; Miller, Alex; Zhang, Xinyan; Lynch, Jessie; Kim, Dennis; Ribeil, Jean-Antoine; Asmal, Mohammed; Goyal, Sunita; Thompson, Alexis A; Tisdale, John F.

N Engl J Med ; 386(7): 617-628, 2022 02 17.

Article in English | MEDLINE | ID: mdl-34898139

3.

Betibeglogene Autotemcel Gene Therapy for Non-ß⁰/ß⁰ Genotype ß-Thalassemia.

Locatelli, Franco; Thompson, Alexis A; Kwiatkowski, Janet L; Porter, John B; Thrasher, Adrian J; Hongeng, Suradej; Sauer, Martin G; Thuret, Isabelle; Lal, Ashutosh; Algeri, Mattia; Schneiderman, Jennifer; Olson, Timothy S; Carpenter, Ben; Amrolia, Persis J; Anurathapan, Usanarat; Schambach, Axel; Chabannon, Christian; Schmidt, Manfred; Labik, Ivan; Elliot, Heidi; Guo, Ruiting; Asmal, Mohammed; Colvin, Richard A; Walters, Mark C.

N Engl J Med ; 386(5): 415-427, 2022 02 03.

Article in English | MEDLINE | ID: mdl-34891223

4.

Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.

Locatelli, Franco; Corbacioglu, Selim; Hobbs, William; Frangoul, Haydar; Walters, Mark C.

Am J Hematol ; 99(3): 430-438, 2024 Mar.

Article in English | MEDLINE | ID: mdl-38010293

5.

Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.

Kanter, Julie; Thompson, Alexis A; Pierciey, Francis J; Hsieh, Matthew; Uchida, Naoya; Leboulch, Philippe; Schmidt, Manfred; Bonner, Melissa; Guo, Ruiting; Miller, Alex; Ribeil, Jean-Antoine; Davidson, David; Asmal, Mohammed; Walters, Mark C; Tisdale, John F.

Am J Hematol ; 98(1): 11-22, 2023 01.

Article in English | MEDLINE | ID: mdl-36161320

6.

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.

Brazauskas, Ruta; Scigliuolo, Graziana M; Wang, Hai-Lin; Cappelli, Barbara; Ruggeri, Annalisa; Fitzhugh, Courtney D; Hankins, Jane S; Kanter, Julie; Meerpohl, Joerg J; Panepinto, Julie A; Rondelli, Damiano; Shenoy, Shalini; Walters, Mark C; Wagner, John E; Tisdale, John F; Gluckman, Eliane; Eapen, Mary.

Blood ; 136(5): 623-626, 2020 07 30.

Article in English | MEDLINE | ID: mdl-32518950

7.

Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.

Thompson, Alexis A; Walters, Mark C; Kwiatkowski, Janet; Rasko, John E J; Ribeil, Jean-Antoine; Hongeng, Suradej; Magrin, Elisa; Schiller, Gary J; Payen, Emmanuel; Semeraro, Michaela; Moshous, Despina; Lefrere, Francois; Puy, Hervé; Bourget, Philippe; Magnani, Alessandra; Caccavelli, Laure; Diana, Jean-Sébastien; Suarez, Felipe; Monpoux, Fabrice; Brousse, Valentine; Poirot, Catherine; Brouzes, Chantal; Meritet, Jean-François; Pondarré, Corinne; Beuzard, Yves; Chrétien, Stany; Lefebvre, Thibaud; Teachey, David T; Anurathapan, Usanarat; Ho, P Joy; von Kalle, Christof; Kletzel, Morris; Vichinsky, Elliott; Soni, Sandeep; Veres, Gabor; Negre, Olivier; Ross, Robert W; Davidson, David; Petrusich, Alexandria; Sandler, Laura; Asmal, Mohammed; Hermine, Olivier; De Montalembert, Mariane; Hacein-Bey-Abina, Salima; Blanche, Stéphane; Leboulch, Philippe; Cavazzana, Marina.

N Engl J Med ; 378(16): 1479-1493, 2018 04 19.

Article in English | MEDLINE | ID: mdl-29669226

8.

Induction of Fetal Hemoglobin by Gene Therapy.

Walters, Mark C.

N Engl J Med ; 384(3): 284-285, 2021 01 21.

Article in English | MEDLINE | ID: mdl-33471981

9.

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Gluckman, Eliane; Cappelli, Barbara; Bernaudin, Francoise; Labopin, Myriam; Volt, Fernanda; Carreras, Jeanette; Pinto Simões, Belinda; Ferster, Alina; Dupont, Sophie; de la Fuente, Josu; Dalle, Jean-Hugues; Zecca, Marco; Walters, Mark C; Krishnamurti, Lakshmanan; Bhatia, Monica; Leung, Kathryn; Yanik, Gregory; Kurtzberg, Joanne; Dhedin, Nathalie; Kuentz, Mathieu; Michel, Gerard; Apperley, Jane; Lutz, Patrick; Neven, Bénédicte; Bertrand, Yves; Vannier, Jean Pierre; Ayas, Mouhab; Cavazzana, Marina; Matthes-Martin, Susanne; Rocha, Vanderson; Elayoubi, Hanadi; Kenzey, Chantal; Bader, Peter; Locatelli, Franco; Ruggeri, Annalisa; Eapen, Mary.

Blood ; 129(11): 1548-1556, 2017 03 16.

Article in English | MEDLINE | ID: mdl-27965196

10.

Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.

Eapen, Mary; Kou, Jianqun; Andreansky, Martin; Bhatia, Monica; Brochstein, Joel; Chaudhury, Sonali; Haight, Ann E; Haines, Hilary; Jacobsohn, David; Jaroscak, Jennifer; Kasow, Kimberly A; Krishnamurti, Lakshmanan; Levine, John E; Leung, Kathryn; Margolis, David; Yu, Lolie C; Horowitz, Mary M; Kamani, Naynesh; Walters, Mark C; Shenoy, Shalini.

Am J Hematol ; 99(4): 785-788, 2024 Apr.

Article in English | MEDLINE | ID: mdl-38343182

11.

Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.

Krishnamurti, Lakshmanan; Neuberg, Donna S; Sullivan, Keith M; Kamani, Naynesh R; Abraham, Allistair; Campigotto, Federico; Zhang, Wandi; Dahdoul, Thabat; De Castro, Laura; Parikh, Suhag; Bakshi, Nitya; Haight, Ann; Hassell, Kathryn L; Loving, Rebekah; Rosenthal, Joseph; Smith, Shannon L; Smith, Wally; Spearman, Marcus; Stevenson, Kristen; Wu, Catherine J; Wiedl, Christina; Waller, Edmund K; Walters, Mark C.

Am J Hematol ; 94(4): 446-454, 2019 04.

Article in English | MEDLINE | ID: mdl-30637784

12.

Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.

Shenoy, Shalini; Gaziev, Javid; Angelucci, Emanuele; King, Allison; Bhatia, Monica; Smith, Angela; Bresters, Dorine; Haight, Anne E; Duncan, Christine N; de la Fuente, Josu; Dietz, Andrew C; Baker, K Scott; Pulsipher, Michael A; Walters, Mark C.

Biol Blood Marrow Transplant ; 24(7): 1313-1321, 2018 07.

Article in English | MEDLINE | ID: mdl-29653206

13.

Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.

Shenoy, Shalini; Walters, Mark C; Ngwube, Alex; Soni, Sandeep; Jacobsohn, David; Chaudhury, Sonali; Grimley, Michael; Chan, Kawah; Haight, Ann; Kasow, Kimberley A; Parikh, Suhag; Andreansky, Martin; Connelly, Jim; Delgado, David; Godder, Kamar; Hale, Gregory; Nieder, Michael; Pulsipher, Michael A; Trachtenberg, Felicia; Neufeld, Ellis; Kwiatkowski, Janet L; Thompson, Alexis A.

Biol Blood Marrow Transplant ; 24(6): 1216-1222, 2018 06.

Article in English | MEDLINE | ID: mdl-29374585

14.

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.

Shenoy, Shalini; Eapen, Mary; Panepinto, Julie A; Logan, Brent R; Wu, Juan; Abraham, Allistair; Brochstein, Joel; Chaudhury, Sonali; Godder, Kamar; Haight, Ann E; Kasow, Kimberly A; Leung, Kathryn; Andreansky, Martin; Bhatia, Monica; Dalal, Jignesh; Haines, Hilary; Jaroscak, Jennifer; Lazarus, Hillard M; Levine, John E; Krishnamurti, Lakshmanan; Margolis, David; Megason, Gail C; Yu, Lolie C; Pulsipher, Michael A; Gersten, Iris; DiFronzo, Nancy; Horowitz, Mary M; Walters, Mark C; Kamani, Naynesh.

Blood ; 128(21): 2561-2567, 2016 11 24.

Article in English | MEDLINE | ID: mdl-27625358

15.

Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease.

Abraham, Allistair; Hsieh, Matthew; Eapen, Mary; Fitzhugh, Courtney; Carreras, Jeanette; Keesler, Daniel; Guilcher, Gregory; Kamani, Naynesh; Walters, Mark C; Boelens, Jaap J; Tisdale, John; Shenoy, Shalini.

Biol Blood Marrow Transplant ; 23(12): 2178-2183, 2017 Dec.

Article in English | MEDLINE | ID: mdl-28882446

16.

The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation for Immune Deficiencies, Inherited Marrow Failure Disorders, and Hemoglobinopathies.

Dietz, Andrew C; Duncan, Christine N; Alter, Blanche P; Bresters, Dorine; Cowan, Morton J; Notarangelo, Luigi; Rosenberg, Philip S; Shenoy, Shalini; Skinner, Roderick; Walters, Mark C; Wagner, John; Baker, K Scott; Pulsipher, Michael A.

Biol Blood Marrow Transplant ; 23(1): 24-29, 2017 01.

Article in English | MEDLINE | ID: mdl-27737772

17.

Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation.

Shenoy, Shalini; Angelucci, Emanuele; Arnold, Staci D; Baker, K Scott; Bhatia, Monica; Bresters, Dorine; Dietz, Andrew C; De La Fuente, Josu; Duncan, Christine; Gaziev, Javid; King, Allison A; Pulsipher, Michael A; Smith, Angela R; Walters, Mark C.

Biol Blood Marrow Transplant ; 23(4): 552-561, 2017 Apr.

Article in English | MEDLINE | ID: mdl-28065838

18.

Sickle marrow: double, double toil and trouble.

Walters, Mark C.

Blood ; 135(23): 2017-2018, 2020 06 04.

Article in English | MEDLINE | ID: mdl-32497220

19.

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.

Walters, Mark C; De Castro, Laura M; Sullivan, Keith M; Krishnamurti, Lakshmanan; Kamani, Naynesh; Bredeson, Christopher; Neuberg, Donna; Hassell, Kathryn L; Farnia, Stephanie; Campbell, Andrew; Petersdorf, Effie.

Biol Blood Marrow Transplant ; 22(2): 207-211, 2016 Feb.

Article in English | MEDLINE | ID: mdl-26500093

20.

Update of hematopoietic cell transplantation for sickle cell disease.

Walters, Mark C.

Curr Opin Hematol ; 22(3): 227-33, 2015 May.

Article in English | MEDLINE | ID: mdl-25767957

VHL CLAP/WR-PAHO/WHO

Latin-American Center for Perinatology, Woman and Reproductive Health

Config filters

Your selection (0)

Export format:

Export:

Exagamglogene Autotemcel for Severe Sickle Cell Disease.

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.

Betibeglogene Autotemcel Gene Therapy for Non-ß⁰/ß⁰ Genotype ß-Thalassemia.

Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.

Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.

Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.

Induction of Fetal Hemoglobin by Gene Therapy.

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.

Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.

Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.

Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.

Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.

Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease.

Sickle marrow: double, double toil and trouble.

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.

Update of hematopoietic cell transplantation for sickle cell disease.