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Journal Articles

First Documentation of Cell-to-Cell Peroxisome Transfer in Hematopoietic Stem and Progenitor Cells: A Method to "Relieve Stress"?

Wilaiwan Durose, Ashish O. Gupta, Paul J. Orchard, Erin Nolan, Ashley C. Kramer, Troy C. Lund

Blood (2022) 140 (Supplement 1): 1686–1687.

https://doi.org/10.1182/blood-2022-155851

Published: 2022

Journal Articles

Bodyweight and Absolute Lymphocyte Count Based Dosing of Rabbit Anti-Thymocyte Globulin Results in Early CD4+ Immune Reconstitution in Patients with Inborn Errors of Metabolism Undergoing Umbilical Cord Blood Transplantation

Daniel Drozdov, Susie L. Long, Ashish O. Gupta, Troy C. Lund, Jaap Jan Boelens, Paul J. Orchard

Blood (2022) 140 (Supplement 1): 4797–4798.

https://doi.org/10.1182/blood-2022-158585

Published: 2022

Journal Articles

Adrenoleukodystrophy Protein (ALDP) Antibody Formation and Graft Loss in Gene Therapy for Adrenoleukodystrophy

Ashish O. Gupta, Paul J. Orchard, Stuti Thakker, Wilaiwan Durose, Carina King, Troy C. Lund

Blood (2022) 140 (Supplement 1): 10641–10642.

https://doi.org/10.1182/blood-2022-155852

Published: 2022

Journal Articles

Phase II Study of Myeloablative 8/8- or 7/8-Matched Allotransplantation with Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil: Marked Reduction in Gvhd Risk without Increased Relapse Risk Compared to Historical Cyclosporine/Methotrexate

Blood (2022) 140 (Supplement 1): 282–283.

https://doi.org/10.1182/blood-2022-165782

Published: 2022

Journal Articles

Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy

Paul J. Orchard, David R. Nascene, Weston P. Miller, Ashish Gupta, Dan Kenney-Jung, Troy C. Lund

Blood (2019) 133 (12): 1378–1381.

https://doi.org/10.1182/blood-2018-11-887240

Published: 2019

Includes: Supplemental data

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

Neurocognitive Trajectory of Patients with Childhood Cerebral Adrenoleukodystrophy Who Received Allogeneic Hematopoietic Cell Transplantation at an Early Stage of Cerebral Disease

Elizabeth Pierpont, PhD, Julie Eisengart, PhD, Richard Ziegler, PhD, Gerald Raymond, Paul J Orchard, Troy Lund, Weston P Miller

Blood (2016) 128 (22): 4682.

https://doi.org/10.1182/blood.V128.22.4682.4682

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

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

Hematopoietic stem cell transplantation for infantile osteopetrosis

Paul J. Orchard, Anders L. Fasth, Jennifer Le Rademacher, Wensheng He, Jaap Jan Boelens, Edwin M. Horwitz, Amal Al-Seraihy, Mouhab Ayas, Carmem M. Bonfim, Farid Boulad, Troy Lund, David K. Buchbinder, Neena Kapoor, Tracey A. O’Brien, Miguel A. Diaz Perez, Paul A. Veys, Mary Eapen

Blood (2015) 126 (2): 270–276.

https://doi.org/10.1182/blood-2015-01-625541

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

Outcomes Following Allogeneic Hematopoietic Stem Cell Transplantation For Metachromatic Leukodystrophy

Alexander A Boucher, MD, Gerald V Raymond, MD, Ryan Shanley, Paul J Orchard, MD, Weston P Miller, IV, MD

Blood (2013) 122 (21): 714.

https://doi.org/10.1182/blood.V122.21.714.714

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

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

Weston P. Miller, Steven M. Rothman, David Nascene, Teresa Kivisto, Todd E. DeFor, Richard S. Ziegler, Julie Eisengart, Kara Leiser, Gerald Raymond, Troy C. Lund, Jakub Tolar, Paul J. Orchard

Blood (2011) 118 (7): 1971–1978.

https://doi.org/10.1182/blood-2011-01-329235

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

Journal Articles

Acid sphingomyelinase deficiency does not protect from graft-versus-host disease in transplant recipients with Niemann-Pick disease

Jakub Tolar, Mary Eapen, Paul J. Orchard, Bruce R. Blazar

Blood (2010) 115 (2): 434–435.

https://doi.org/10.1182/blood-2009-09-245167

Published: 2010

Journal Articles

Patient-Specific Induced Pluripotent Stem Cells in Hurler Syndrome

Jakub Tolar, MD, PhD, In-Hyun Park, M.D., Lily Xia, B.S., Mark Osborn, B.S., Ron T. McElmurry, B.S., Paul J. Orchard, M.D., George Q. Daley, MD, PhD, Bruce R Blazar, M.D.

Blood (2008) 112 (11): 386.

https://doi.org/10.1182/blood.V112.11.386.386

Published: 2008

Journal Articles

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Jakub Tolar, MD, PhD, Paul J. Orchard, M.D., Nigel Key, M.D., Bruce R. Blazar, M.D.

Blood (2007) 110 (11): 2160.5.

https://doi.org/10.1182/blood.V110.11.2160.5.2160.5

Published: 2007

Journal Articles

Dyskeratosis Congenita: Low Regimen-Related Toxicity Following Hematopoietic Cell Transplantation (HCT) Using a Reduced Intensity Conditioning Regimen.

Jakub Tolar, MD, PhD, Paul J. Orchard, M.D., Jeffrey S. Miller, M.D., Margaret L. MacMillan, M.D., John E. Wagner, M.D., Blanche P. Alter, M.D., MPH, K.S. Baker, M.D., M.S.

Blood (2007) 110 (11): 2005.

https://doi.org/10.1182/blood.V110.11.2005.2005

Published: 2007

Journal Articles

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Jakub Tolar, MD, PhD, Paul J. Orchard, M.D., Nigel Key, M.D., Bruce R. Blazar, M.D.

Blood (2007) 110 (11): 3951.

https://doi.org/10.1182/blood.V110.11.3951.3951

Published: 2007

NARROW

First Documentation of Cell-to-Cell Peroxisome Transfer in Hematopoietic Stem and Progenitor Cells: A Method to "Relieve Stress"?

Bodyweight and Absolute Lymphocyte Count Based Dosing of Rabbit Anti-Thymocyte Globulin Results in Early CD4+ Immune Reconstitution in Patients with Inborn Errors of Metabolism Undergoing Umbilical Cord Blood Transplantation

Adrenoleukodystrophy Protein (ALDP) Antibody Formation and Graft Loss in Gene Therapy for Adrenoleukodystrophy

Phase II Study of Myeloablative 8/8- or 7/8-Matched Allotransplantation with Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil: Marked Reduction in Gvhd Risk without Increased Relapse Risk Compared to Historical Cyclosporine/Methotrexate

Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy

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

Neurocognitive Trajectory of Patients with Childhood Cerebral Adrenoleukodystrophy Who Received Allogeneic Hematopoietic Cell Transplantation at an Early Stage of Cerebral Disease

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

Hematopoietic stem cell transplantation for infantile osteopetrosis

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

Outcomes Following Allogeneic Hematopoietic Stem Cell Transplantation For Metachromatic Leukodystrophy

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

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

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

Acid sphingomyelinase deficiency does not protect from graft-versus-host disease in transplant recipients with Niemann-Pick disease

Patient-Specific Induced Pluripotent Stem Cells in Hurler Syndrome

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

Dyskeratosis Congenita: Low Regimen-Related Toxicity Following Hematopoietic Cell Transplantation (HCT) Using a Reduced Intensity Conditioning Regimen.

Glycosaminoglycans as Anticoagulants in Mucopolysaccharidosis Type I (MPS I).

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American Society of Hematology

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American Society of Hematology

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