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Journal Articles

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

Kevin A. Goncalves, PhD, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Hans J. Hertzler, Anthony E. Boitano, PhD, Michael P. Cooke, PhD

Blood (2019) 134 (Supplement_1): 4424.

https://doi.org/10.1182/blood-2019-131189

Published: 2019

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

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

First Results of a Prospective Multicenter Phase I/II Clinical Trial in Adult Patients Using Tcralpha/Beta and CD19 Depleted Haploidentical Stem Cell Transplantation Following Reduced Intensity Conditioning

Wolfgang Bethge, MD, Stephan Mielke, MD, Dietger Niederwieser, MD, Roland Meisel, MD, Donald w. Bunjes, MD, Arne Brecht, MD, Jurgen Kuball, MD, Sandra Karitzky, PhD, Silke Holtkamp, Christiane Siewert, PhD, Stefanie Pflitsch, Michael Schumm, DVM, Matthias K. Eyrich, MD, Vladan Vucinic, Markus Wiesneth, MD, Kasper Westinga, Rupert Handgretinger, MD PhD

Blood (2017) 130 (Supplement 1): 213.

https://doi.org/10.1182/blood.V130.Suppl_1.213.213

Published: 2017

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

The Changing Patterns of Graft Failure in MPS1H, Hurler Syndrome: A Review of 30-Years Experience

Su Han Lum, Weston P Miller, Simon Jones, Jean Mercer, Troy Lund, Denise Bonney, Paul J Orchard, Robert Wynn

Blood (2016) 128 (22): 4700.

https://doi.org/10.1182/blood.V128.22.4700.4700

Published: 2016

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

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

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

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

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

Long-term outcomes in MPS-IH: throwing stars

Michael A. Pulsipher

Blood (2015) 125 (13): 2016–2017.

https://doi.org/10.1182/blood-2015-02-626507

Published: 2015

Journal Articles

Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I

Alice Pievani, Isabella Azario, Laura Antolini, Tsutomu Shimada, Pravin Patel, Cristina Remoli, Benedetta Rambaldi, Maria Grazia Valsecchi, Mara Riminucci, Andrea Biondi, Shunji Tomatsu, Marta Serafini

Blood (2015) 125 (10): 1662–1671.

https://doi.org/10.1182/blood-2014-06-581207

Published: 2015

Includes: Supplemental data

Journal Articles

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

Alice Pievani, PhD, Isabella Maria Rebecca Azario, Laura Antolini, Tsutomu Shimada, Pravin Patel, Cristina Remoli, Benedetta Rambaldi, Maria Grazia Valsecchi, Mara Riminucci, Andrea Biondi, Shunji Tomatsu, Marta Serafini

Blood (2014) 124 (21): 649.

https://doi.org/10.1182/blood.V124.21.649.649

Published: 2014

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

Platelets: An Efficient Depot for Generation and Distribution of Lysosomal Idua in Enzyme-Deficient MPS I Mice.

Mei Dai, PhD, Phuong Cao, Dao Pan, PhD

Blood (2012) 120 (21): 3157.

https://doi.org/10.1182/blood.V120.21.3157.3157

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

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

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

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

Jakub Tolar, In-Hyun Park, Lily Xia, Chris J. Lees, Brandon Peacock, Beau Webber, Ron T. McElmurry, Cindy R. Eide, Paul J. Orchard, Michael Kyba, Mark J. Osborn, Troy C. Lund, John E. Wagner, George Q. Daley, Bruce R. Blazar

Blood (2011) 117 (3): 839–847.

https://doi.org/10.1182/blood-2010-05-287607

Published: 2011

Includes: Supplemental data

NARROW

High Dose Hematopoietic Stem Cell Transplantation Leads to Rapid Hematopoietic and Microglia Recovery and Disease Correction in a Mouse Model of Hurler Syndrome

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

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

First Results of a Prospective Multicenter Phase I/II Clinical Trial in Adult Patients Using Tcralpha/Beta and CD19 Depleted Haploidentical Stem Cell Transplantation Following Reduced Intensity Conditioning

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

The Changing Patterns of Graft Failure in MPS1H, Hurler Syndrome: A Review of 30-Years Experience

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

AAV Gene Therapy for MPS1 Corneal Clouding and Joint Stiffness

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

Lysosomal Newborn Screening in a Cohort in Mexican Population-

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

Long-term outcomes in MPS-IH: throwing stars

Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I

Neonatal Bone Marrow Transplantation Prevents Bone Pathology in a Mouse Model of Mucopolysaccharidosis Type I

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

Platelets: An Efficient Depot for Generation and Distribution of Lysosomal Idua in Enzyme-Deficient MPS I Mice.

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

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

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

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

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American Society of Hematology

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American Society of Hematology

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