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2020
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation
Polgreen, L. E., Lund, T. C., Braunlin, E., Tolar, J., Miller, B. S., Fung, E., Whitley, C. B., Eisengart, J. B., Northrop, E., Rudser, K., Miller, W. P. & Orchard, P. J., Jan 1 2020, In: Pediatric Research. 87, 1, p. 104-111 8 p.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology
Hampe, C. S., Eisengart, J. B., Lund, T. C., Orchard, P. J., Swietlicka, M., Wesley, J. & McIvor, R. S., Aug 5 2020, In: Cells. 9, 8Research output: Contribution to journal › Review article › peer-review
Open Access -
Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders
Gupta, A., Downey, M., Shanley, R., Jennissen, C., Miller, W. P., Lund, T. C., Orchard, P. J. & Smith, A. R., Mar 2020, In: Biology of Blood and Marrow Transplantation. 26, 3, p. 486-492 7 p.Research output: Contribution to journal › Article › peer-review
2 Scopus citations -
2019
Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter
Braunlin, E. A., Miettunen, K., Lund, T. C., Luquette, M. & Orchard, P. J., Feb 2019, In: Molecular Genetics and Metabolism. 126, 2, p. 117-120 4 p.Research output: Contribution to journal › Article › peer-review
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Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes
Eisengart, J. B., Pierpont, E. I., Kaizer, A. M., Rudser, K. D., King, K. E., Pasquali, M., Polgreen, L. E., Dickson, P. I., Le, S. Q., Miller, W. P., Tolar, J., Orchard, P. J. & Lund, T. C., Nov 1 2019, In: Genetics in Medicine. 21, 11, p. 2552-2560 9 p.Research output: Contribution to journal › Article › peer-review
Open Access6 Scopus citations -
Late Mortality after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A Report from the Blood or Marrow Transplant Survivor Study-2 (BMTSS-2)
Wadhwa, A., Chen, Y., Holmqvist, A., Wu, J., Ness, E., Parman, M., Kung, M., Hageman, L., Francisco, L., Braunlin, E., Miller, W., Lund, T., Armenian, S., Arora, M., Orchard, P. & Bhatia, S., Feb 2019, In: Biology of Blood and Marrow Transplantation. 25, 2, p. 328-334 7 p.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes
Lund, T. C., Miller, W. P., Liao, A. Y., Tolar, J., Shanley, R., Pasquali, M., Sando, N., Bigger, B. W., Polgreen, L. E. & Orchard, P. J., Dec 1 2019, In: Scientific reports. 9, 1, 14105.Research output: Contribution to journal › Article › peer-review
Open Access1 Scopus citations -
Recommendations for the management of MPS IVA: Systematic evidence- A nd consensus-based guidance
Akyol, M. U., Alden, T. D., Amartino, H., Ashworth, J., Belani, K., Berger, K. I., Borgo, A., Braunlin, E., Eto, Y., Gold, J. I., Jester, A., Jones, S. A., Karsli, C., Mackenzie, W., Marinho, D. R., McFadyen, A., McGill, J., Mitchell, J. J., Muenzer, J., Okuyama, T. & 9 others, , Jun 13 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 137.Research output: Contribution to journal › Article › peer-review
Open Access13 Scopus citations -
Recommendations for the management of MPS VI: Systematic evidence- and consensus-based guidance
Akyol, M. U., Alden, T. D., Amartino, H., Ashworth, J., Belani, K., Berger, K. I., Borgo, A., Braunlin, E., Eto, Y., Gold, J. I., Jester, A., Jones, S. A., Karsli, C., Mackenzie, W., Marinho, D. R., McFadyen, A., McGill, J., Mitchell, J. J., Muenzer, J., Okuyama, T. & 9 others, , May 29 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 118.Research output: Contribution to journal › Article › peer-review
Open Access5 Scopus citations -
Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: Results of an international consensus procedure
Kuiper, G. A., Langereis, E. J., Breyer, S., Carbone, M., Castelein, R. M., Eastwood, D. M., Garin, C., Guffon, N., Van Hasselt, P. M., Hensman, P., Jones, S. A., Kenis, V., Kruyt, M., Van Der Lee, J. H., Mackenzie, W. G., Orchard, P. J., Oxborrow, N., Parini, R., Robinson, A., Schubert Hjalmarsson, E. & 2 others, , Jan 18 2019, In: Orphanet Journal of Rare Diseases. 14, 1, 17.Research output: Contribution to journal › Article › peer-review
Open Access3 Scopus citations -
2018
Erratum: Publisher Correction: Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1 (Scientific reports (2018) 8 1 (3681))
Makino, E., Klodnitsky, H., Leonard, J., Lillie, J., Lund, T. C., Marshall, J., Nietupski, J., Orchard, P. J., Miller, W. P., Phaneuf, C., Tietz, D., Varban, M. L., Donovan, M. & Belenki, A., Mar 19 2018, In: Scientific reports. 8, 1, p. 4994 1 p., 4994.Research output: Contribution to journal › Comment/debate › peer-review
Open Access -
Fast, sensitive method for trisaccharide biomarker detection in mucopolysaccharidosis type 1
Makino, E., Klodnitsky, H., Leonard, J., Lillie, J., Lund, T. C., Marshall, J., Nietupski, J., Orchard, P. J., Miller, W. P., Phaneuf, C., Tietz, D., Varban, M. L., Donovan, M. & Belenki, A., Dec 1 2018, In: Scientific reports. 8, 1, 3681.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
Eisengart, J. B., Rudser, K. D., Xue, Y., Orchard, P., Miller, W., Lund, T., Van der Ploeg, A., Mercer, J., Jones, S., Mengel, K. E., Gökce, S., Guffon, N., Giugliani, R., de Souza, C. F. M., Shapiro, E. G. & Whitley, C. B., Nov 1 2018, In: Genetics in Medicine. 20, 11, p. 1423-1429 7 p.Research output: Contribution to journal › Article › peer-review
Open Access20 Scopus citations -
Metabolic Syndrome and Cardiovascular Risk Factors after Hematopoietic Cell Transplantation in Severe Mucopolysaccharidosis Type I (Hurler Syndrome)
Braunlin, E., Steinberger, J., DeFor, T., Orchard, P. & Kelly, A. S., Jun 2018, In: Biology of Blood and Marrow Transplantation. 24, 6, p. 1289-1293 5 p.Research output: Contribution to journal › Article › peer-review
Open Access -
2017
Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: Potential for biomarker development and insight into pathogenesis
Hinderer, C., Katz, N., Louboutin, J. P., Bell, P., Tolar, J., Orchard, P. J., Lund, T. C., Nayal, M., Weng, L., Mesaros, C., de Souza, C. F. M., Corte, A. D., Giugliani, R. & Wilson, J. M., Oct 1 2017, In: Human molecular genetics. 26, 19, p. 3837-3849 13 p.Research output: Contribution to journal › Article › peer-review
Open Access4 Scopus citations -
Allele-Level HLA Matching Impacts Key Outcomes Following Umbilical Cord Blood Transplantation for Inherited Metabolic Disorders
Mallhi, K. K., Smith, A. R., DeFor, T. E., Lund, T. C., Orchard, P. J. & Miller, W. P., Jan 1 2017, In: Biology of Blood and Marrow Transplantation. 23, 1, p. 119-125 7 p.Research output: Contribution to journal › Article › peer-review
Open Access21 Scopus citations -
Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome
Lum, S. H., Miller, W. P., Jones, S., Poulton, K., Ogden, W., Lee, H., Logan, A., Bonney, D., Lund, T. C., Orchard, P. J. & Wynn, R. F., Jun 1 2017, In: Bone marrow transplantation. 52, 6, p. 846-853 8 p.Research output: Contribution to journal › Article › peer-review
16 Scopus citations -
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, L. R., Temme, A., Podetz-Pedersen, K. M., Riedl, M., Vulchanova, L., Robinson, N., Hanson, L. R., Kozarsky, K. F., Orchard, P. J., Frey, W. H., Low, W. C. & Scott McIvor, R., Jul 2017, In: Human gene therapy. 28, 7, p. 576-587 12 p.Research output: Contribution to journal › Article › peer-review
Open Access19 Scopus citations -
Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience
Rodgers, N. J., Kaizer, A. M., Miller, W. P., Rudser, K., Orchard, P. J. & Braunlin, E. A., Mar 1 2017, In: Journal of Inherited Metabolic Disease. 40, 2, p. 271-280 10 p.Research output: Contribution to journal › Article › peer-review
17 Scopus citations -
The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison
Wyffels, M. L., Orchard, P. J., Shanley, R. M., Miller, W. P. & Van Heest, A. E., Jul 2017, In: Journal of Hand Surgery. 42, 7, p. 573.e1-573.e8Research output: Contribution to journal › Article › peer-review
4 Scopus citations -
Treatment of brain disease in the mucopolysaccharidoses
Scarpa, M., Orchard, P. J., Schulz, A., Dickson, P. I., Haskins, M. E., Escolar, M. L. & Giugliani, R., Dec 2017, In: Molecular Genetics and Metabolism. 122, p. 25-34 10 p.Research output: Contribution to journal › Review article › peer-review
Open Access28 Scopus citations -
2016
Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H
Raymond, G. V., Pasquali, M., Polgreen, L. E., Dickson, P. I., Miller, W. P., Orchard, P. J. & Lund, T. C., Dec 2 2016, In: Scientific reports. 6, 38305.Research output: Contribution to journal › Article › peer-review
Open Access7 Scopus citations -
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres
Ghosh, A., Miller, W., Orchard, P. J., Jones, S. A., Mercer, J., Church, H. J., Tylee, K., Lund, T., Bigger, B. W., Tolar, J. & Wynn, R. F., Mar 1 2016, In: Molecular Genetics and Metabolism. 117, 3, p. 373-377 5 p.Research output: Contribution to journal › Article › peer-review
Open Access34 Scopus citations -
Health-related quality of life in mucopolysaccharidosis: Looking beyond biomedical issues
Hendriksz, C. J., Berger, K. I., Lampe, C., Kircher, S. G., Orchard, P. J., Southall, R., Long, S., Sande, S. & Gold, J. I., Aug 26 2016, In: Orphanet Journal of Rare Diseases. 11, 1, 119.Research output: Contribution to journal › Review article › peer-review
Open Access15 Scopus citations -
Long-term cognitive and functional outcomes in children with mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) treated with hematopoietic cell transplantation
Kunin-Batson, A. S., Shapiro, E. G., Rudser, K., Lavery, C. A., Bjoraker, K. J., Jones, S. A., Wynn, R. F., Vellodi, A., Tolar, J., Orchard, P. J. & Wraith, J. E., 2016, JIMD Reports. Springer, p. 95-102 8 p. (JIMD Reports; vol. 29).Research output: Chapter in Book/Report/Conference proceeding › Chapter
Open Access20 Scopus citations -
Progression of hip dysplasia in mucopolysaccharidosis type ihurler after successful hematopoietic stem cell transplantation
Langereis, E. J., Den Os, M. M., Breen, C., Jones, S. A., Knaven, O. C., Mercer, J., Miller, W. P., Kelly, P. M., Kennedy, J., Ketterl, T. G., Omeara, A., Orchard, P. J., Lund, T. C., Van Rijn, R. R., Sakkers, R. J., White, K. K. & Wijburg, F. A., Mar 2 2016, In: Journal of Bone and Joint Surgery - American Volume. 98, 5, p. 386-395 10 p.Research output: Contribution to journal › Article › peer-review
13 Scopus citations -
Unique medical issues in adult patients with mucopolysaccharidoses
Mitchell, J., Berger, K. I., Borgo, A., Braunlin, E. A., Burton, B. K., Ghotme, K. A., Kircher, S. G., Molter, D., Orchard, P. J., Palmer, J., Pastores, G. M., Rapoport, D. M., Wang, R. Y. & White, K., Oct 1 2016, In: European Journal of Internal Medicine. 34, p. 2-10 9 p.Research output: Contribution to journal › Review article › peer-review
Open Access15 Scopus citations -
2015
Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: An international multicenter study
Aldenhoven, M., Wynn, R. F., Orchard, P. J., O'Meara, A., Veys, P., Fischer, A., Valayannopoulos, V., Neven, B., Rovelli, A., Prasad, V. K., Tolar, J., Allewelt, H., Jones, S. A., Parini, R., Renard, M., Bordon, V., Wulffraat, N. M., De Koning, T. J., Shapiro, E. G., Kurtzberg, J. & 1 others, , Mar 26 2015, In: Blood. 125, 13, p. 2164-2172 9 p.Research output: Contribution to journal › Article › peer-review
149 Scopus citations -
Long-term results of carpal tunnel and trigger finger releases in a patient with Hurler syndrome
Wyffels, M., Orchard, P. J. & Van Heest, A. E., Jan 28 2015, In: JBJS case connector. 5, 1, e27.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment
Shapiro, E. G., Nestrasil, I., Rudser, K., Delaney, K., Kovac, V., Ahmed, A., Yund, B., Orchard, P. J., Eisengart, J., Niklason, G. R., Raiman, J., Mamak, E., Cowan, M. J., Bailey-Olson, M., Harmatz, P., Shankar, S. P., Cagle, S., Ali, N., Steiner, R. D., Wozniak, J. & 2 others, , Sep 1 2015, In: Molecular Genetics and Metabolism. 116, 1-2, p. 61-68 8 p.Research output: Contribution to journal › Article › peer-review
35 Scopus citations -
2014
Biomarkers of bone remodeling in children with mucopolysaccharidosis types I, II, and VI
Stevenson, D. A., Rudser, K., Kunin-Batson, A., Fung, E. B., Viskochil, D., Shapiro, E., Orchard, P. J., Whitley, C. B. & Polgreen, L. E., 2014, In: Journal of Pediatric Rehabilitation Medicine. 7, 2, p. 159-165 7 p.Research output: Contribution to journal › Article › peer-review
15 Scopus citations -
Effect of recombinant human growth hormone on changes in height, bone mineral density, and body composition over 1-2years in children with Hurler or Hunter syndrome
Polgreen, L. E., Thomas, W., Orchard, P. J., Whitley, C. B. & Miller, B. S., Jan 1 2014, In: Molecular Genetics and Metabolism. 111, 2, p. 101-106 6 p.Research output: Contribution to journal › Article › peer-review
10 Scopus citations -
Isokinetic muscle strength differences in patients with mucopolysaccharidosis I, II, and VI
Taylor, N. E., Dengel, D. R., Lund, T. C., Rudser, K. D., Orchard, P. J., Steinberger, J., Whitley, C. B. & Polgreen, L. E., 2014, In: Journal of Pediatric Rehabilitation Medicine. 7, 4, p. 353-360 8 p.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
Low bone mineral content and challenges in interpretation of dual-energy X-ray absorptiometry in children with mucopolysaccharidosis types I, II, and VI
Polgreen, L. E., Thomas, W., Fung, E., Viskochil, D., Stevenson, D. A., Steinberger, J., Orchard, P. J., Whitley, C. B. & Ensrud, K. E., Jan 2014, In: Journal of Clinical Densitometry. 17, 1, p. 200-206 7 p.Research output: Contribution to journal › Article › peer-review
13 Scopus citations -
Unexpected coronary artery findings in mucopolysaccharidosis. Report of four cases and literature review
Braunlin, E. A., Orchard, P. J., Whitley, C. B., Schroeder, L., Reed, R. C. & Manivel, J. C., Jan 1 2014, In: Cardiovascular Pathology. 23, 3, p. 145-151 7 p.Research output: Contribution to journal › Article › peer-review
15 Scopus citations -
2013
Cardiac ultrasound findings in infants with severe (hurler phenotype) untreated mucopolysaccharidosis (MPS) type i
Schroeder, L., Orchard, P., Whitley, C. B., Berry, J. M., Tolar, J., Miller, W. & Braunlin, E. A., 2013, JIMD Reports. Springer, p. 87-94 8 p. (JIMD Reports; vol. 10).Research output: Chapter in Book/Report/Conference proceeding › Chapter
7 Scopus citations -
Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome
Eisengart, J. B., Rudser, K. D., Tolar, J., Orchard, P. J., Kivisto, T., Ziegler, R. S., Whitley, C. B. & Shapiro, E. G., Feb 2013, In: Journal of Pediatrics. 162, 2, p. 375-380.e1Research output: Contribution to journal › Article › peer-review
40 Scopus citations -
Hip dysplasia in patients with hurler syndrome (mucopolysaccharidosis type 1H)
Thawrani, D. P., Walker, K., Polgreen, L. E., Tolar, J. & Orchard, P. J., Sep 1 2013, In: Journal of Pediatric Orthopaedics. 33, 6, p. 635-643 9 p.Research output: Contribution to journal › Article › peer-review
15 Scopus citations -
Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning
Boelens, J. J., Aldenhoven, M., Purtill, D., Ruggeri, A., Defor, T., Wynn, R., Wraith, E., Cavazzana-Calvo, M., Rovelli, A., Fischer, A., Tolar, J., Prasad, V. K., Escolar, M., Gluckman, E., O'Meara, A., Orchard, P. J., Veys, P., Eapen, M., Kurtzberg, J., Rocha, V. & 4 others, , May 9 2013, In: Blood. 121, 19, p. 3981-3987 7 p.Research output: Contribution to journal › Article › peer-review
126 Scopus citations -
2012
An exploratory study of brain function and structure in mucopolysaccharidosis type I: Long term observations following hematopoietic cell transplantation (HCT)
Shapiro, E., Guler, O. E., Rudser, K., Delaney, K., Bjoraker, K., Whitley, C., Tolar, J., Orchard, P., Provenzale, J. & Thomas, K. M., Sep 2012, In: Molecular Genetics and Metabolism. 107, 1-2, p. 116-121 6 p.Research output: Contribution to journal › Article › peer-review
26 Scopus citations -
Heparan sulfate and dermatan sulfate derived disaccharides are sensitive markers for newborn screening for mucopolysaccharidoses types I, II and III
de Ruijter, J., de Ru, M. H., Wagemans, T., IJlst, L., Lund, A. M., Orchard, P. J., Schaefer, G. B., Wijburg, F. A. & van Vlies, N., Dec 1 2012, In: Molecular Genetics and Metabolism. 107, 4, p. 705-710 6 p.Research output: Contribution to journal › Article › peer-review
70 Scopus citations -
2011
Analysis of glycosaminoglycans in cerebrospinal fluid from patients with mucopolysaccharidoses by isotope-dilution ultra-performance liquid chromatography - Tandem mass spectrometry
Zhang, H., Young, S. P., Auray-Blais, C., Orchard, P. J., Tolar, J. & Millington, D. S., Jul 2011, In: Clinical chemistry. 57, 7, p. 1005-1012 8 p.Research output: Contribution to journal › Article › peer-review
59 Scopus citations -
Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
Tolar, J., Park, I. H., Xia, L., Lees, C. J., Peacock, B., Webber, B., McElmurry, R. T., Eide, C. R., Orchard, P. J., Kyba, M., Osborn, M. J., Lund, T. C., Wagner, J. E., Daley, G. Q. & Blazar, B. R., Jan 20 2011, In: Blood. 117, 3, p. 839-847 9 p.Research output: Contribution to journal › Article › peer-review
66 Scopus citations -
Outcome of penetrating keratoplasty for mucopolysaccharidoses
Bothun, E. D., Decanini, A., Summers, C. G., Orchard, P. J. & Tolar, J., Feb 2011, In: Archives of Ophthalmology. 129, 2, p. 138-144 7 p.Research output: Contribution to journal › Article › peer-review
12 Scopus citations -
2010
Pre-transplant risk factors affecting outcome in Hurler syndrome
Orchard, P. J., Milla, C., Braunlin, E., Defor, T., Bjoraker, K., Blazar, B. R., Peters, C., Wagner, J. & Tolar, J., Jul 2010, In: Bone marrow transplantation. 45, 7, p. 1239-1246 8 p.Research output: Contribution to journal › Article › peer-review
22 Scopus citations -
2009
Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I
Tolar, J., Braunlin, E., Riddle, M., Peacock, B., McElmurry, R. T., Orchard, P. J. & Blazar, B. R., Sep 2009, In: Journal of Heart Valve Disease. 18, 5, p. 524-529 6 p.Research output: Contribution to journal › Article › peer-review
6 Scopus citations -
Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation
Polgreen, L. E., Plog, M., Schwender, J. D., Tolar, J., Thomas, W., Orchard, P. J., Miller, B. S. & Petryk, A., 2009, In: Bone marrow transplantation. 44, 5, p. 279-285 7 p.Research output: Contribution to journal › Article › peer-review
22 Scopus citations -
2008
Circulating anticoagulant glycosaminoglycans in mucopolysaccharidosis type I
Tolar, J., Orchard, P. J., Key, N. S. & Blazar, B. R., May 2008, In: Journal of Thrombosis and Haemostasis. 6, 5, p. 893-895 3 p.Research output: Contribution to journal › Article › peer-review
5 Scopus citations -
Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome
Tolar, J., Grewal, S. S., Bjoraker, K. J., Whitley, C. B., Shapiro, E. G., Charnas, L. & Orchard, P. J., Mar 2008, In: Bone marrow transplantation. 41, 6, p. 531-535 5 p.Research output: Contribution to journal › Article › peer-review
101 Scopus citations -
Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation
Polgreen, L. E., Tolar, J., Plog, M., Himes, J. H., Orchard, P. J., Whitley, C. B., Miller, B. S. & Petryk, A., Jun 2008, In: Bone marrow transplantation. 41, 12, p. 1005-1011 7 p.Research output: Contribution to journal › Article › peer-review
58 Scopus citations