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Wu, Calvin C, Econs, Michael J, DiMeglio, Linda A, Insogna, Karl L, Levine, Michael A, Orchard, Paul J, Miller, Weston P, Petryk, Anna, Rush, Eric T, Shoback, Dolores M, Ward, Leanne M, Polgreen, Lynda E
Published in The journal of clinical endocrinology and metabolism (01.09.2017)
Published in The journal of clinical endocrinology and metabolism (01.09.2017)
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Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report
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Published in Orphanet journal of rare diseases (07.08.2015)
Published in Orphanet journal of rare diseases (07.08.2015)
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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
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Published in The New England journal of medicine (26.10.2017)
Published in The New England journal of medicine (26.10.2017)
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Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
Aldenhoven, Mieke, Wynn, Robert F., Orchard, Paul J., O’Meara, Anne, Veys, Paul, Fischer, Alain, Valayannopoulos, Vassili, Neven, Benedicte, Rovelli, Attilio, Prasad, Vinod K., Tolar, Jakub, Allewelt, Heather, Jones, Simon A., Parini, Rossella, Renard, Marleen, Bordon, Victoria, Wulffraat, Nico M., de Koning, Tom J., Shapiro, Elsa G., Kurtzberg, Joanne, Boelens, Jaap Jan
Published in Blood (26.03.2015)
Published in Blood (26.03.2015)
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Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report
Miller, Weston P., Rothman, Steven M., Nascene, David, Kivisto, Teresa, DeFor, Todd E., Ziegler, Richard S., Eisengart, Julie, Leiser, Kara, Raymond, Gerald, Lund, Troy C., Tolar, Jakub, Orchard, Paul J.
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Published in Blood (18.08.2011)
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Differences in MPS I and MPS II Disease Manifestations
Hampe, Christiane S., Yund, Brianna D., Orchard, Paul J., Lund, Troy C., Wesley, Jacob, McIvor, R. Scott
Published in International journal of molecular sciences (23.07.2021)
Published in International journal of molecular sciences (23.07.2021)
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TCIRG1 Transgenic Rescue of Osteoclast Function Using Induced Pluripotent Stem Cells Derived from Patients with Infantile Malignant Autosomal Recessive Osteopetrosis
Chen, Weili, Twaroski, Kirk, Eide, Cindy, Riddle, Megan J, Orchard, Paul J, Tolar, Jakub
Published in Journal of bone and joint surgery. American volume (06.11.2019)
Published in Journal of bone and joint surgery. American volume (06.11.2019)
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A report on state‐wide implementation of newborn screening for X‐linked Adrenoleukodystrophy
Wiens, Katie, Berry, Susan A., Choi, Hyoung, Gaviglio, Amy, Gupta, Ashish, Hietala, Amy, Kenney‐Jung, Daniel, Lund, Troy, Miller, Weston, Pierpont, Elizabeth I., Raymond, Gerald, Winslow, Holly, Zierhut, Heather A., Orchard, Paul J.
Published in American journal of medical genetics. Part A (01.07.2019)
Published in American journal of medical genetics. Part A (01.07.2019)
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Transplantation in inborn errors of metabolism: current considerations and future perspectives
Boelens, Jaap Jan, Orchard, Paul J., Wynn, Robert F.
Published in British journal of haematology (01.11.2014)
Published in British journal of haematology (01.11.2014)
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Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion
Lund, Troy C., Orchard, Paul J., Nascene, David R., King, Carina J., Braun, Jennifer, Thakkar, Stuti, Durose, Willa, Shestopalov, Ilya, Thakar, Himal, Gupta, Ashish O.
Published in Molecular therapy (05.08.2024)
Published in Molecular therapy (05.08.2024)
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Treatment of brain disease in the mucopolysaccharidoses
Scarpa, Maurizio, Orchard, Paul J., Schulz, Angela, Dickson, Patricia I., Haskins, Mark E., Escolar, Maria L., Giugliani, Roberto
Published in Molecular genetics and metabolism (01.12.2017)
Published in Molecular genetics and metabolism (01.12.2017)
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Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Gupta, Ashish O, Raymond, Gerald, Pierpont, Elizabeth I, Kemp, Stephan, McIvor, R Scott, Rayannavar, Arpana, Miller, Bradley, Lund, Troy C, Orchard, Paul J
Published in Expert opinion on biological therapy (02.09.2022)
Published in Expert opinion on biological therapy (02.09.2022)
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Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10‐year prospective study
Miller, Bradley S., Fung, Ellen B., White, Klane K., Lund, Troy C., Harmatz, Paul, Orchard, Paul J., Whitley, Chester B., Polgreen, Lynda E.
Published in Journal of inherited metabolic disease (01.07.2023)
Published in Journal of inherited metabolic disease (01.07.2023)
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Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy
Orchard, Paul J., Nascene, David R., Miller, Weston P., Gupta, Ashish, Kenney-Jung, Dan, Lund, Troy C.
Published in Blood (21.03.2019)
Published in Blood (21.03.2019)
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MECHANISMS OF DISEASE: Osteopetrosis
Tolar, Jakub, Teitelbaum, Steven L, Orchard, Paul J
Published in The New England journal of medicine (30.12.2004)
Published in The New England journal of medicine (30.12.2004)
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Hematopoietic stem cell transplantation for infantile osteopetrosis
Orchard, Paul J., Fasth, Anders L., Le Rademacher, Jennifer, He, Wensheng, Boelens, Jaap Jan, Horwitz, Edwin M., Al-Seraihy, Amal, Ayas, Mouhab, Bonfim, Carmem M., Boulad, Farid, Lund, Troy, Buchbinder, David K., Kapoor, Neena, O'Brien, Tracey A., Perez, Miguel A. Diaz, Veys, Paul A., Eapen, Mary
Published in Blood (09.07.2015)
Published in Blood (09.07.2015)
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Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
Belur, Lalitha R, Temme, Alexa, Podetz-Pedersen, Kelly M, Riedl, Maureen, Vulchanova, Lucy, Robinson, Nicholas, Hanson, Leah R, Kozarsky, Karen F, Orchard, Paul J, Frey, 2nd, William H, Low, Walter C, McIvor, R Scott
Published in Human gene therapy (01.07.2017)
Published in Human gene therapy (01.07.2017)
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Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance
Akyol, Mehmet Umut, Alden, Tord D, Amartino, Hernan, Ashworth, Jane, Belani, Kumar, Berger, Kenneth I, Borgo, Andrea, Braunlin, Elizabeth, Eto, Yoshikatsu, Gold, Jeffrey I, Jester, Andrea, Jones, Simon A, Karsli, Cengiz, Mackenzie, William, Marinho, Diane Ruschel, McFadyen, Andrew, McGill, Jim, Mitchell, John J, Muenzer, Joseph, Okuyama, Torayuki, Orchard, Paul J, Stevens, Bob, Thomas, Sophie, Walker, Robert, Wynn, Robert, Giugliani, Roberto, Harmatz, Paul, Hendriksz, Christian, Scarpa, Maurizio
Published in Orphanet journal of rare diseases (13.06.2019)
Published in Orphanet journal of rare diseases (13.06.2019)
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