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Journal Articles

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Bernhard Gentner, MD, Maria Ester Bernardo, MD PhD, Francesca Tucci, MD, Erika Zonari, PhD, Francesca Fumagalli, MD, Silvia Pontesilli, MD, Serena Acquati, PhD, Paolo Silvani, MD, Fabio Ciceri, MD, Attilio Rovelli, MD, Giancarlo La Marca, PhD, Rossella Parini, MD, Silvia Gregori, PhD, Eugenio Montini, PhD, Luigi Naldini, MD PhD, Alessandro Aiuti, MD PhD

Blood (2019) 134 (Supplement_1): 607.

https://doi.org/10.1182/blood-2019-128805

Published: 2019

Journal Articles

Immune Mediated Cytopenia's Following Cord Blood Transplantation for Hurler's Syndrome: A Failure of Pre-Transplant Immune Suppression

David Deambrosis, MBBS, FRACP, Ryan Hum, Prashant Hiwarkar, Kay Poulton, Denise Bonney, Helen Jane Campbell, FRCP, MD FRCPath, Robert Wynn, MD

Blood (2018) 132 (Supplement 1): 3374.

https://doi.org/10.1182/blood-2018-99-112995

Published: 2018

Journal Articles

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34 + Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

Paul Orchard, MD, Glen D. Raffel, MD, Carolyn H Condon, MPH, Catherine A Monaghan, Demetra Vernet, Suzanne Tracey Sheirr, Jennifer Braun, RN BSN, Ryan Shanley, M.S, Troy C Lund, MD MS, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, John C Davis, MD MPH, John E. Wagner, MD

Blood (2018) 132 (Supplement 1): 3467.

https://doi.org/10.1182/blood-2018-99-115102

Published: 2018

Journal Articles

Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders

Michael Downey, BA, Ashish O. Gupta, MBBS, MPH, Ryan Shanley, M.S, Weston P. Miller, MD, Paul Orchard, MD, Angela R. Smith, MD MS

Blood (2018) 132 (Supplement 1): 3352.

https://doi.org/10.1182/blood-2018-99-117573

Published: 2018

Journal Articles

Hematopoietic Stem Cell Transplantation for Children with Neurological Diseases Improves the Outcomes and Not Leads to Significant Late Effects — Report from Russian Group

Kirill Kirgizov, MD PhD, Anastasia Borovkova, MD, Elena Skorobogatova, MD PhD, Tatjana Bykova, MD PhD, Alexey A Maschan, MD PhD Professor, Ludmila S Zubarovskaya, MD PhD Professor, Axel R. Zander, MD PhD Professor, Boris V Afanasyev, MD PhD Professor, Alexander Rumyantsev, MD PhD Professor

Blood (2017) 130 (Supplement 1): 4526.

https://doi.org/10.1182/blood.V130.Suppl_1.4526.4526

Published: 2017

Journal Articles

High-Exposure, Targeted Daily Busulfan and Fludarabine-Based Conditioning for Children Undergoing Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders: Outcomes at a Single Center

Weston P Miller, Melisa Kay Stricherz, PharmD, Cathryn Jennissen, PharmD, Nicole Sando, Jennifer Danielson, Troy Lund, Paul J Orchard

Blood (2016) 128 (22): 2191.

https://doi.org/10.1182/blood.V128.22.2191.2191

Published: 2016

Journal Articles

Patient and Parental Perspectives on Long-Term Adaptive, Behavioral and Functional Outcomes Following Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders

Rachel Phelan, MD MPH, Paul J Orchard, MD, Margaret Semrud-Clikeman, PhD, Nicholas Smiley, Weston P Miller, IV, MD

Blood (2015) 126 (23): 4351.

https://doi.org/10.1182/blood.V126.23.4351.4351

Published: 2015

Journal Articles

Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders

Carter Biewen, BA, Angela R Smith, MD MS, Jakub Tolar, MD, PhD, Weston P Miller, IV, MD

Blood (2015) 126 (23): 1969.

https://doi.org/10.1182/blood.V126.23.1969.1969

Published: 2015

Journal Articles

Lysosomal Newborn Screening in a Cohort in Mexican Population-

Juana Ines Navarrete, MD

Blood (2015) 126 (23): 5581.

https://doi.org/10.1182/blood.V126.23.5581.5581

Published: 2015

Journal Articles

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

Melisa A. Vance, PhD, Matthew Hirsch, PhD, Richard Jude Samulski, PhD, Joanne Kurtzberg, MD, Laurie Goodrich, DVM, PhD ACVS Diplomate, Llanga Telmo, Bsc

Blood (2015) 126 (23): 2041.

https://doi.org/10.1182/blood.V126.23.2041.2041

Published: 2015

Journal Articles

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Blood (2015) 125 (13): 2164–2172.

https://doi.org/10.1182/blood-2014-11-608075

Published: 2015

Includes: Supplemental data

Journal Articles

Risk Factors for Graft Failure with Busulfan/Fludarabine-Based Conditioning in Children Undergoing Allogeneic Hematopoietic Cell Transplantation for Nonmalignant Disorders

Benjamin Huang, MD, Christopher C. Dvorak, MD, Justin Wahlstrom, MD, Jimmy Hwang, PhD, Janel R Long-Boyle, PharmD, PhD, Morton Jerome Cowan, MD, Biljana N. Horn, MD

Blood (2014) 124 (21): 1155.

https://doi.org/10.1182/blood.V124.21.1155.1155

Published: 2014

Journal Articles

Better BMT for Hurler syndrome—on the level?

Michael A. Pulsipher

Blood (2013) 121 (19): 3785–3787.

https://doi.org/10.1182/blood-2013-04-491779

Published: 2013

Journal Articles

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

Blood (2013) 121 (19): 3981–3987.

https://doi.org/10.1182/blood-2012-09-455238

Published: 2013

Journal Articles

Cytokine Environement Analysis During Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases

Fabien Touzot, MD, PhD, Bryan Fiema, Sophie Paczesny, M.D., Ph.D, Isabelle Andre-Schmutz, PhD, Dominique Helley, MD, PhD, Despina Moshous, MD, PhD, Corinne De Chappedelaine, Stephane Blanche, MD, Alain Fischer, MD/PhD, Marina Cavazzana-Calvo, MD, PhD

Blood (2012) 120 (21): 4484.

https://doi.org/10.1182/blood.V120.21.4484.4484

Published: 2012

Journal Articles

Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

Mieke Aldenhoven, Maria Escolar, Robert Wynn, MRCP, FRCPath, MD, Ed Wraith, Anne O'Meara, Paul Veys, Nizar Mahlaoui, MD, Michele Poe, Paul Orchard, MD, Joanne Kurtzberg, MD, Jaap J Boelens, MD, PhD

Blood (2012) 120 (21): 1958.

https://doi.org/10.1182/blood.V120.21.1958.1958

Published: 2012

Journal Articles

Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I,

H. Angharad Watson, PhD, Rebecca J Holley, PhD, Kia J Langford-Smith, Fiona L Wilkinson, PhD, Toin H van Kuppevelt, Robert Wynn, MD, J. Ed Wraith, Catherine L R Merry, Brian W Bigger, PhD

Blood (2011) 118 (21): 4010.

https://doi.org/10.1182/blood.V118.21.4010.4010

Published: 2011

Journal Articles

Correction of Immune and Enzyme Deficiency by Bone Marrow Transplant in PNP Deficient SCID

Vikramajit Singh, MRCP, Robert Wynn, MD, Denise Bonney, PhD, Stephen Hughes, MD, Kay Poulton, PhD, Helena Lee, PhD

Blood (2011) 118 (21): 4556.

https://doi.org/10.1182/blood.V118.21.4556.4556

Published: 2011

Journal Articles

Successful Long- Term Outcome and Chimeric Status Following Allogeneic Stem Cell Transplantation for Non-Malignant Disorders Using Moderate T Cell Depletion with No Post Transplant Gvhd Prophylaxis.

Ronit Elhasid, MD, Irina Zaidman, MD, Khalil Abdalla, MD, Miriam Ben-Arush, MD, Nuhad Haddad, MD, Tsila Zuckerman, MD, Osnat Gidoni, Dina Rubin, Rina Katz, Shimon Polack, MD, Yair Reisner, Ph.D, Jacob M. Rowe, MD

Blood (2008) 112 (11): 3292.

https://doi.org/10.1182/blood.V112.11.3292.3292

Published: 2008

Journal Articles

Pulmonary Function in Pediatric survivors of non-Malignant Disorders after Allogeneic Hematopoietic Stem Cell Transplantation.

Megan S Motosue, Hiroto Inaba, MD, PhD, Jianmin Pan, PhD, Dennis C. Stokes, MD, Deo Kumar Srivastava, PhD, Kimberly A. Kasow, DO, Gregory A. Hale, MD, Wing Leung, MD, PhD

Blood (2008) 112 (11): 2136.

https://doi.org/10.1182/blood.V112.11.2136.2136

Published: 2008

NARROW

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

Immune Mediated Cytopenia's Following Cord Blood Transplantation for Hurler's Syndrome: A Failure of Pre-Transplant Immune Suppression

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34 + Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders

Hematopoietic Stem Cell Transplantation for Children with Neurological Diseases Improves the Outcomes and Not Leads to Significant Late Effects — Report from Russian Group

High-Exposure, Targeted Daily Busulfan and Fludarabine-Based Conditioning for Children Undergoing Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders: Outcomes at a Single Center

Patient and Parental Perspectives on Long-Term Adaptive, Behavioral and Functional Outcomes Following Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders

Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders

Lysosomal Newborn Screening in a Cohort in Mexican Population-

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Risk Factors for Graft Failure with Busulfan/Fludarabine-Based Conditioning in Children Undergoing Allogeneic Hematopoietic Cell Transplantation for Nonmalignant Disorders

Better BMT for Hurler syndrome—on the level?

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

Cytokine Environement Analysis During Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases

Long Term Outcomes of Hematopoietic Stem Cell Transplantation in Patients with Severe Phenotype Hurler Syndrome: an International Multi-Center Study

Excess Heparan Sulphate Inhibits CXCL12-Mediated Hematopoietic Cell Migration and Engraftment After Bone Marrow Transplant in Mice with Mucopolysaccharidosis Type I,

Correction of Immune and Enzyme Deficiency by Bone Marrow Transplant in PNP Deficient SCID

Successful Long- Term Outcome and Chimeric Status Following Allogeneic Stem Cell Transplantation for Non-Malignant Disorders Using Moderate T Cell Depletion with No Post Transplant Gvhd Prophylaxis.

Pulmonary Function in Pediatric survivors of non-Malignant Disorders after Allogeneic Hematopoietic Stem Cell Transplantation.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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