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Journal Articles

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Tanja C. Vallée, Jannik S. Glasmacher, Hannes Buchner, Peter D. Arkwright, Uta Behrends, Anastasia Bondarenko, Michael J. Browning, David Buchbinder, Alessandro Cattoni, Liudmyla Chernyshova, Peter Ciznar, Theresa Cole, Wojciech Czogała, Gregor Dueckers, John David M. Edgar, Fatih Erbey, Anders Fasth, Francesca Ferrua, Renata Formankova, Eleonora Gambineri, Andrew R. Gennery, Frederick D. Goldman, Luis I. Gonzalez-Granado, Carsten Heilmann, Tarja Heiskanen-Kosma, Hanna Juntti, Leena Kainulainen, Hirokazu Kanegane, Neslihan E. Karaca, Sara S. Kilic, Christoph Klein, Sylwia Kołtan, Irina Kondratenko, Isabelle Meyts, Gulnara M. Nasrullayeva, Lucia D. Notarangelo, Srdjan Pasic, Isabelle Pellier, Claudio Pignata, Siraj Misbah, Ansgar Schulz, Gesmar R. Segundo, Anna Shcherbina, Mary Slatter, Robert Sokolic, Pere Soler-Palacin, Polina Stepensky, Joris M. van Montfrans, Samppa Ryhänen, Beata Wolska-Kuśnierz, John B. Ziegler, Xiaodong Zhao, Alessandro Aiuti, Hans D. Ochs, Michael H. Albert

Blood (2024) 143 (24): 2504–2516.

https://doi.org/10.1182/blood.2023021411

Published: 2024

Includes: Supplemental data

Journal Articles

Alternative Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease in Europe

Blood (2018) 132 (Supplement 1): 4645.

https://doi.org/10.1182/blood-2018-99-111129

Published: 2018

Journal Articles

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

Jannik S Glasmacher, Tanja C Bittner, MD, Hans D Ochs, MD, Alessandro Aiuti, MD PhD, Peter D Arkwright, MD, Dmitry Balashov, Uta Behrends, Bernd H. Belohradsky, MD, Elisa Bertoni, David K. Buchbinder, MD MSHS, Michael Browning, Anastasiia Bondarenko, Fabio Candotti, MD, Alessandro Cattoni, Liudmyla Chernyshova, Joseph H. Chewning, MD, Peter Ciznar, Theresa Cole, Beatriz T Costa-Carvalho, Wojciech Czogala, Gregor Dueckers, David M Edgar, Fatih Erbey, Anders Fasth, MD PhD, Renata Formankova, MD PhD, Tomas Freiberger, Eleonora Gambineri, Andrew Gennery, MD, Frederick D Goldman, MD, Luis I Gonzalez-Granado, Tiago N Gulmaraes, David Hagin, MD PhD, Fabian Hauck, MD PhD, Tarja Heiskanen-Kosma, Manfred Hoenig, MD, Hanna Juntti, Hirokazu Kanegane, Leena Kainulainen, Neslihan E Karaca, Sara S Kilic, Christoph Klein, MD PhD, Sylwia Koltan, Irina Kondratenko, Dawei Liu, MD, Susanne Matthes, MD, Juliana T L Mazzucchelli, Isabelle Meyts, Siraj Misbah, Zohreh Nademi, Gulnara Nasrullayeva, Lucia D Notarangelo, Pere Soler-Palacin, Olga Pashchenko, Srdjan Pasic, Isabelle Pellier, MD, Claudio Pignata, Camilla Roepstorff, Catharina Schuetz, Ansgar S Schulz, MD PhD Prof., Gesmar R S Segundo, Anna Shcherbina, Joanne Smart, Robert A. Sokolic, MD, Polina Stepensky, MD, Troy Torgerson, MD PhD, Svetlana Vakhlyarskaya, Joris van Montfrans, MD PhD, Kim Vettenranta, MD PD, Beata Wolska-Kusnierz, Xiaodong Zhao, John B Ziegler, Xuan Zhang, Michael H. Albert, MD

Blood (2016) 128 (22): 366.

https://doi.org/10.1182/blood.V128.22.366.366

Published: 2016

Journal Articles

Neonatal screening for severe primary immunodeficiency diseases using high-throughput triplex real-time PCR

Stephan Borte, Ulrika von Döbeln, Anders Fasth, Ning Wang, Magdalena Janzi, Jacek Winiarski, Ulrich Sack, Qiang Pan-Hammarström, Michael Borte, Lennart Hammarström

Blood (2012) 119 (11): 2552–2555.

https://doi.org/10.1182/blood-2011-08-371021

Published: 2012

Includes: Supplemental data

Journal Articles

Gene Transfer to CD34+ Cells From Peripheral Blood Restores Osteoclast Function in Infantile Malignant Osteopetrosis

Ilana Moscatelli, PhD, Christian Thudium, Maria Askmyr, PhD, Ansgar S Schulz, MD, PhD, Oscar Porras, MD, PhD, Anders Fasth, MD, PhD, Kim Henriksen, PhD, Johan Richter, MD, PhD

Blood (2011) 118 (21): 2058.

https://doi.org/10.1182/blood.V118.21.2058.2058

Published: 2011

Journal Articles

Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party

Blood (2011) 118 (6): 1693–1698.

https://doi.org/10.1182/blood-2011-02-336156

Published: 2011

Journal Articles

Incidence and Risk Factors for Secondary Autoimmune Diseases (AD) After Hematopoietic Stem Cell Transplantation (HSCT) for a Severe Autoimmune Disease-A Retrospective EBMT WP AD Study

Blood (2010) 116 (21): 2322.

https://doi.org/10.1182/blood.V116.21.2322.2322

Published: 2010

Journal Articles

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

Blood (2010) 115 (16): 3231–3238.

https://doi.org/10.1182/blood-2009-09-239087

Published: 2010

Includes: Supplemental data

Journal Articles

Long-Term Survival and Late Deaths After Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Diseases and Inborn Errors of Metabolism.

Blood (2009) 114 (22): 3320.

https://doi.org/10.1182/blood.V114.22.3320.3320

Published: 2009

Journal Articles

Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Blood (2008) 111 (1): 439–445.

https://doi.org/10.1182/blood-2007-03-076679

Published: 2008

Includes: Supplemental data

Journal Articles

Hematopoietic stem cell–targeted neonatal gene therapy reverses lethally progressive osteopetrosis in oc/oc mice

Maria K. Johansson, Teun J. de Vries, Ton Schoenmaker, Mats Ehinger, Ann C. M. Brun, Anders Fasth, Stefan Karlsson, Vincent Everts, Johan Richter

Blood (2007) 109 (12): 5178–5185.

https://doi.org/10.1182/blood-2006-12-061382

Published: 2007

Journal Articles

Hematopoietic Stem Cell Transplantation Cures Oc/Oc Mice from Osteopetrosis.

Maria Johansson, Anders Fasth, Stefan Karlsson, Johan Richter

Blood (2004) 104 (11): 2140.

https://doi.org/10.1182/blood.V104.11.2140.2140

Published: 2004

Journal Articles

Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002

Blood (2004) 103 (3): 1152–1157.

https://doi.org/10.1182/blood-2003-06-2014

Published: 2004

Journal Articles

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

Blood (2001) 97 (6): 1598–1603.

https://doi.org/10.1182/blood.V97.6.1598

Published: 2001

Journal Articles

V(D)J recombination defects in lymphocytes due to RAG mutations: severe immunodeficiency with a spectrum of clinical presentations

Blood (2001) 97 (1): 81–88.

https://doi.org/10.1182/blood.V97.1.81

Published: 2001

Journal Articles

Long-Term Immune Reconstitution and Outcome After HLA-Nonidentical T-Cell–Depleted Bone Marrow Transplantation for Severe Combined Immunodeficiency: A European Retrospective Study of 116 Patients

Elie Haddad, Paul Landais, Wilhelm Friedrich, Bert Gerritsen, M. Cavazzana-Calvo, Gareth Morgan, Yves Bertrand, Anders Fasth, Fulvio Porta, Andrew Cant, Theresa Espanol, Susannah Müller, Paul Veys, Jaak Vossen, Alain Fischer

Blood (1998) 91 (10): 3646–3653.

https://doi.org/10.1182/blood.V91.10.3646

Published: 1998

NARROW

Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Alternative Donor Hematopoietic Stem Cell Transplantation for Sickle Cell Disease in Europe

Wiskott-Aldrich Syndrome: A Retrospective Study on 575 Patients Analyzing the Impact of Splenectomy, Stem Cell Transplantation, or No Definitive Treatment on Frequency of Disease-Related Complications and Physician-Perceived Quality of Life

Neonatal screening for severe primary immunodeficiency diseases using high-throughput triplex real-time PCR

Gene Transfer to CD34+ Cells From Peripheral Blood Restores Osteoclast Function in Infantile Malignant Osteopetrosis

Secondary autoimmune diseases occurring after HSCT for an autoimmune disease: a retrospective study of the EBMT Autoimmune Disease Working Party

Incidence and Risk Factors for Secondary Autoimmune Diseases (AD) After Hematopoietic Stem Cell Transplantation (HSCT) for a Severe Autoimmune Disease-A Retrospective EBMT WP AD Study

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

Long-Term Survival and Late Deaths After Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Diseases and Inborn Errors of Metabolism.

Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Hematopoietic stem cell–targeted neonatal gene therapy reverses lethally progressive osteopetrosis in oc/oc mice

Hematopoietic Stem Cell Transplantation Cures Oc/Oc Mice from Osteopetrosis.

Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002

Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program

V(D)J recombination defects in lymphocytes due to RAG mutations: severe immunodeficiency with a spectrum of clinical presentations

Long-Term Immune Reconstitution and Outcome After HLA-Nonidentical T-Cell–Depleted Bone Marrow Transplantation for Severe Combined Immunodeficiency: A European Retrospective Study of 116 Patients

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American Society of Hematology

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ASH Publications

American Society of Hematology

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