TY - JOUR AU - Muenzer, J. AU - Wraith, J. E. AU - Clarke, L. A. PY - 2009 DA - 2009// TI - International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines JO - Pediatrics VL - 123 UR - https://doi.org/10.1542/peds.2008-0416 DO - 10.1542/peds.2008-0416 ID - Muenzer2009 ER - TY - JOUR AU - Muenzer, J. PY - 2011 DA - 2011// TI - Overview of the mucopolysaccharidoses JO - Rheumatology VL - 50 UR - https://doi.org/10.1093/rheumatology/ker394 DO - 10.1093/rheumatology/ker394 ID - Muenzer2011 ER - TY - JOUR AU - Beck, M. AU - Arn, P. AU - Giugliani, R. PY - 2014 DA - 2014// TI - The natural history of MPS I: global perspectives from the MPS I Registry JO - Genet Med VL - 16 UR - https://doi.org/10.1038/gim.2014.25 DO - 10.1038/gim.2014.25 ID - Beck2014 ER - TY - JOUR AU - Moore, D. AU - Connock, M. J. AU - Wraith, E. AU - Lavery, C. PY - 2008 DA - 2008// TI - The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler–Scheie and Scheie syndromes in the UK JO - Orphanet J Rare Dis VL - 16 UR - https://doi.org/10.1186/1750-1172-3-24 DO - 10.1186/1750-1172-3-24 ID - Moore2008 ER - TY - JOUR AU - Clarke, L. A. AU - Giugliani, R. AU - Guffon, N. PY - 2019 DA - 2019// TI - Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): insights from the International MPS I Registry JO - Clin Genet VL - 96 UR - https://doi.org/10.1111/cge.13583 DO - 10.1111/cge.13583 ID - Clarke2019 ER - TY - JOUR AU - Guffon, N. AU - Souillet, G. AU - Maire, I. AU - Straczek, J. AU - Guibaud, P. PY - 1998 DA - 1998// TI - Follow-up of nine patients with Hurler syndrome after bone marrow transplantation JO - J Pediatr VL - 133 UR - https://doi.org/10.1016/S0022-3476(98)70201-X DO - 10.1016/S0022-3476(98)70201-X ID - Guffon1998 ER - TY - JOUR AU - Souillet, G. AU - Guffon, N. AU - Maire, I. AU - Pujol, M. PY - 2003 DA - 2003// TI - Outcome of 27 patients with Hurler’s syndrome transplanted from either related or unrelated haematopoietic stem cell sources JO - Bone Marrow Transplant VL - 31 UR - https://doi.org/10.1038/sj.bmt.1704105 DO - 10.1038/sj.bmt.1704105 ID - Souillet2003 ER - TY - JOUR AU - Tomatsu, S. AU - Alméciga-Díaz, C. J. AU - Montaño, A. M. PY - 2015 DA - 2015// TI - Therapies for the bone in mucopolysaccharidoses JO - Mol Genet Metab VL - 114 UR - https://doi.org/10.1016/j.ymgme.2014.12.001 DO - 10.1016/j.ymgme.2014.12.001 ID - Tomatsu2015 ER - TY - JOUR AU - Wraith, J. E. PY - 1995 DA - 1995// TI - The mucopolysaccharidoses: a clinical review and guide to management JO - Arch Dis Child VL - 72 UR - https://doi.org/10.1136/adc.72.3.263 DO - 10.1136/adc.72.3.263 ID - Wraith1995 ER - TY - JOUR AU - Staba, S. L. AU - Escolar, M. L. AU - Poe, M. AU - Kim, Y. PY - 2004 DA - 2004// TI - Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome JO - N Engl J Med VL - 350 UR - https://doi.org/10.1056/NEJMoa032613 DO - 10.1056/NEJMoa032613 ID - Staba2004 ER - TY - JOUR AU - Boelens, J. J. AU - Aldenhoven, M. AU - Purtill, D. AU - Ruggeri, A. PY - 2013 DA - 2013// TI - Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning JO - Blood VL - 121 UR - https://doi.org/10.1182/blood-2012-09-455238 DO - 10.1182/blood-2012-09-455238 ID - Boelens2013 ER - TY - JOUR AU - Peters, C. AU - Shapiro, E. G. AU - Anderson, J. AU - Henslee-Downey, P. J. PY - 1998 DA - 1998// TI - Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group JO - Blood VL - 91 UR - https://doi.org/10.1182/blood.V91.7.2601 DO - 10.1182/blood.V91.7.2601 ID - Peters1998 ER - TY - JOUR AU - Taylor, M. AU - Khan, S. AU - Stapleton, M. PY - 2019 DA - 2019// TI - Hematopoietic stem cell transplantation for mucopolysaccharidoses: past, present, and future JO - Biol Blood Marrow Transplant VL - 25 UR - https://doi.org/10.1016/j.bbmt.2019.02.012 DO - 10.1016/j.bbmt.2019.02.012 ID - Taylor2019 ER - TY - JOUR AU - Ru, M. H. AU - Boelens, J. J. AU - Das, A. M. PY - 2011 DA - 2011// TI - Enzyme replacement therapy and, or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure JO - Orphanet J Rare Dis VL - 6 UR - https://doi.org/10.1186/1750-1172-6-55 DO - 10.1186/1750-1172-6-55 ID - Ru2011 ER - TY - JOUR AU - Sifuentes, M. AU - Doroshow, R. AU - Hoft, R. AU - Mason, G. PY - 2007 DA - 2007// TI - A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years JO - Mol Genet Metab VL - 90 UR - https://doi.org/10.1016/j.ymgme.2006.08.007 DO - 10.1016/j.ymgme.2006.08.007 ID - Sifuentes2007 ER - TY - JOUR AU - Ghosh, A. AU - Miller, W. AU - Orchard, P. J. AU - Jones, S. A. PY - 2016 DA - 2016// TI - Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10-year combined experience of 2 centres JO - Mol Genet Metab VL - 117 UR - https://doi.org/10.1016/j.ymgme.2016.01.011 DO - 10.1016/j.ymgme.2016.01.011 ID - Ghosh2016 ER - TY - JOUR AU - Shapiro, E. G. AU - Nestrasil, I. AU - Rudser, K. PY - 2015 DA - 2015// TI - Neurocognition across the spectrum of mucopolysaccharidosis type I: age, severity, and treatment JO - Mol Genet Metab VL - 116 UR - https://doi.org/10.1016/j.ymgme.2015.06.002 DO - 10.1016/j.ymgme.2015.06.002 ID - Shapiro2015 ER - TY - JOUR AU - Eisengart, J. B. AU - Rudser, K. D. AU - Tolar, J. AU - Orchard, P. J. PY - 2013 DA - 2013// TI - Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome JO - J Pediatr VL - 162 UR - https://doi.org/10.1016/j.jpeds.2012.07.052 DO - 10.1016/j.jpeds.2012.07.052 ID - Eisengart2013 ER - TY - JOUR AU - Aldenhoven, M. AU - Wynn, R. F. AU - Orchard, P. J. AU - O’Meara, A. PY - 2015 DA - 2015// TI - Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study JO - Blood VL - 125 UR - https://doi.org/10.1182/blood-2014-11-608075 DO - 10.1182/blood-2014-11-608075 ID - Aldenhoven2015 ER - TY - JOUR AU - Cox-Brinkman, J. AU - Boelens, J. J. AU - Wraith, J. E. AU - O’meara, A. PY - 2006 DA - 2006// TI - Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome JO - Bone Marrow Transplant VL - 38 UR - https://doi.org/10.1038/sj.bmt.1705401 DO - 10.1038/sj.bmt.1705401 ID - Cox-Brinkman2006 ER - TY - JOUR AU - Poe, M. D. AU - Chagnon, S. L. AU - Escolar, M. L. PY - 2014 DA - 2014// TI - Early treatment is associated with improved cognition in hurler syndrome JO - Ann Neurol VL - 76 UR - https://doi.org/10.1002/ana.24246 DO - 10.1002/ana.24246 ID - Poe2014 ER - TY - JOUR AU - Parini, R. AU - Deodato, F. AU - Rocco, M. AU - Lanino, E. PY - 2017 DA - 2017// TI - Open issues in Mucopolysaccharidosis type I-Hurler JO - Orphanet J Rare Dis VL - 12 UR - https://doi.org/10.1186/s13023-017-0662-9 DO - 10.1186/s13023-017-0662-9 ID - Parini2017 ER - TY - JOUR AU - Yasuda, E. AU - Mackenzie, W. AU - Ruhnke, K. AU - Shimada, T. PY - 2015 DA - 2015// TI - Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements JO - Mol Genet Metab Rep VL - 2 UR - https://doi.org/10.1016/j.ymgmr.2014.12.006 DO - 10.1016/j.ymgmr.2014.12.006 ID - Yasuda2015 ER - TY - JOUR AU - Aldenhoven, M. AU - Jones, S. A. AU - Bonney, D. PY - 2015 DA - 2015// TI - Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines JO - Biol Blood Marrow Transplant VL - 21 UR - https://doi.org/10.1016/j.bbmt.2015.02.011 DO - 10.1016/j.bbmt.2015.02.011 ID - Aldenhoven2015 ER - TY - JOUR AU - Prasad, V. K. AU - Kurtzberg, J. PY - 2010 DA - 2010// TI - Transplant outcomes in mucopolysaccharidoses JO - Semin Hematol VL - 47 UR - https://doi.org/10.1053/j.seminhematol.2009.10.008 DO - 10.1053/j.seminhematol.2009.10.008 ID - Prasad2010 ER - TY - JOUR AU - Linden, M. H. AU - Kruyt, M. C. AU - Sakkers, R. J. AU - Koning, T. J. PY - 2011 DA - 2011// TI - Orthopaedic management of Hurler’s disease after hematopoietic stem cell transplantation: a systematic review JO - J Inherit Metab Dis VL - 34 UR - https://doi.org/10.1007/s10545-011-9304-x DO - 10.1007/s10545-011-9304-x ID - Linden2011 ER - TY - JOUR AU - Shapiro, E. G. AU - Whitley, C. B. AU - Eisengart, J. B. PY - 2018 DA - 2018// TI - Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome JO - Orphanet J Rare Dis VL - 13 UR - https://doi.org/10.1186/s13023-018-0817-3 DO - 10.1186/s13023-018-0817-3 ID - Shapiro2018 ER - TY - JOUR AU - Aldenhoven, M. AU - Boelens, J. J. AU - Koning, T. J. PY - 2008 DA - 2008// TI - The clinical outcome of Hurler syndrome after stem cell transplantation JO - Biol Blood Marrow Transplant VL - 14 UR - https://doi.org/10.1016/j.bbmt.2008.01.009 DO - 10.1016/j.bbmt.2008.01.009 ID - Aldenhoven2008 ER - TY - JOUR AU - Polgreen, L. E. AU - Lund, T. C. AU - Braunlin, E. AU - Tolar, J. PY - 2019 DA - 2019// TI - Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation JO - Pediatr Res UR - https://doi.org/10.1038/s41390-019-0541-2 DO - 10.1038/s41390-019-0541-2 ID - Polgreen2019 ER - TY - JOUR AU - Langereis, E. J. AU - Borgo, A. AU - Crushell, E. AU - Harmatz, P. R. PY - 2013 DA - 2013// TI - Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure JO - Orphanet J Rare Dis VL - 8 UR - https://doi.org/10.1186/1750-1172-8-155 DO - 10.1186/1750-1172-8-155 ID - Langereis2013 ER - TY - JOUR AU - Yuan, C. AU - Bothun, E. D. AU - Hardten, D. R. AU - Tolar, J. AU - McLoon, L. K. PY - 2016 DA - 2016// TI - A novel explanation of corneal clouding in a bone marrow transplant-treated patient with Hurler syndrome JO - Exp Eye Res VL - 148 UR - https://doi.org/10.1016/j.exer.2016.05.022 DO - 10.1016/j.exer.2016.05.022 ID - Yuan2016 ER - TY - JOUR AU - Broek, B. T. A. AU - Smit, A. L. AU - Boelens, J. J. AU - Hasselt, P. M. PY - 2020 DA - 2020// TI - Hearing loss in patients with mucopolysaccharidoses-1 and -6 after hematopoietic cell transplantation: a longitudinal analysis [published online ahead of print, 2020 Jun 24] JO - J Inherit Metab Dis UR - https://doi.org/10.1002/jimd.12277 DO - 10.1002/jimd.12277 ID - Broek2020 ER - TY - JOUR AU - Eisengart, J. B. AU - Rudser, K. D. AU - Xue, Y. AU - Orchard, P. PY - 2018 DA - 2018// TI - Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison JO - Genet Med UR - https://doi.org/10.1038/gim.2018.29 DO - 10.1038/gim.2018.29 ID - Eisengart2018 ER - TY - JOUR AU - Vellodi, A. AU - Young, E. P. AU - Cooper, A. AU - Wraith, J. E. PY - 1997 DA - 1997// TI - Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres JO - Arch Dis Child VL - 76 UR - https://doi.org/10.1136/adc.76.2.92 DO - 10.1136/adc.76.2.92 ID - Vellodi1997 ER - TY - JOUR AU - Escolar, M. L. AU - Jones, S. A. AU - Shapiro, E. G. AU - Horovitz, D. D. G. PY - 2017 DA - 2017// TI - Practical management of behavioral problems in mucopolysaccharidoses disorders JO - Mol Genet Metab VL - 122 UR - https://doi.org/10.1016/j.ymgme.2017.09.010 DO - 10.1016/j.ymgme.2017.09.010 ID - Escolar2017 ER - TY - JOUR AU - Lehtonen, A. AU - Rust, S. AU - Jones, S. AU - Brown, R. AU - Hare, D. PY - 2018 DA - 2018// TI - Social functioning and behaviour in mucopolysaccharidosis IH [Hurlers syndrome] JO - JIMD Rep VL - 39 UR - https://doi.org/10.1007/8904_2017_47 DO - 10.1007/8904_2017_47 ID - Lehtonen2018 ER - TY - JOUR AU - Soni-Jaiswal, A. AU - Mercer, J. AU - Jones, S. A. AU - Bruce, I. A. AU - Callery, P. PY - 2016 DA - 2016// TI - Mucopolysaccharidosis I; Parental beliefs about the impact of disease on the quality of life of their children JO - Orphanet J Rare Dis VL - 11 UR - https://doi.org/10.1186/s13023-016-0478-z DO - 10.1186/s13023-016-0478-z ID - Soni-Jaiswal2016 ER - TY - JOUR AU - Kunin-Batson, A. S. AU - Shapiro, E. G. AU - Rudser, K. D. AU - Lavery, C. A. PY - 2016 DA - 2016// TI - Long-term cognitive and functional outcomes in children with mucopolysaccharidosis (MPS)-IH (Hurler syndrome) treated with hematopoietic cell transplantation JO - JIMD Rep VL - 29 UR - https://doi.org/10.1007/8904_2015_521 DO - 10.1007/8904_2015_521 ID - Kunin-Batson2016 ER - TY - JOUR AU - Shapiro, E. G. AU - Escolar, M. L. AU - Delaney, K. A. AU - Mitchell, J. J. PY - 2017 DA - 2017// TI - Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses JO - Mol Genet Metab VL - 122S UR - https://doi.org/10.1016/j.ymgme.2017.09.007 DO - 10.1016/j.ymgme.2017.09.007 ID - Shapiro2017 ER - TY - JOUR AU - Shapiro, E. G. AU - Jones, S. A. AU - Escolar, M. L. PY - 2017 DA - 2017// TI - Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations—neurological signs and symptoms JO - Mol Genet Metab VL - 122S UR - https://doi.org/10.1016/j.ymgme.2017.08.009 DO - 10.1016/j.ymgme.2017.08.009 ID - Shapiro2017 ER - TY - JOUR AU - Aldenhoven, M. AU - Broek, B. T. A. AU - Wynn, R. F. AU - O’Meara, A. PY - 2017 DA - 2017// TI - Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation JO - Blood Adv VL - 1 UR - https://doi.org/10.1182/bloodadvances.2017011387 DO - 10.1182/bloodadvances.2017011387 ID - Aldenhoven2017 ER - TY - STD TI - Kubaski F, de Oliveira Poswar F, Michelin-Tirelli K, Matte UDS, et al. Mucopolysaccharidosis Type I. Diagnostics (Basel). 2020;10(3):161. https://doi.org/10.3390/diagnostics10030161. ID - ref42 ER - TY - JOUR AU - Seto, T. AU - Kono, K. AU - Morimoto, K. AU - Inoue, Y. PY - 2001 DA - 2001// TI - Brain magnetic resonance imaging in 23 patients with mucopolysaccharidoses and the effect of bone marrow transplantation JO - Ann Neurol VL - 50 UR - https://doi.org/10.1002/ana.1098 DO - 10.1002/ana.1098 ID - Seto2001 ER - TY - JOUR AU - Braunlin, E. A. AU - Harmatz, P. R. AU - Scarpa, M. AU - Furlanetto, B. PY - 2011 DA - 2011// TI - Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management JO - J Inherit Metab Dis VL - 34 UR - https://doi.org/10.1007/s10545-011-9359-8 DO - 10.1007/s10545-011-9359-8 ID - Braunlin2011 ER - TY - JOUR AU - Lum, S. H. AU - Jones, S. A. AU - Ghosh, A. AU - Bigger, B. W. AU - Wynn, R. F. PY - 2016 DA - 2016// TI - Hematopoietic stem cell transplant for the mucopolysaccharidoses JO - Expert Opin Orphan Drugs UR - https://doi.org/10.1517/21678707.2016.1147948 DO - 10.1517/21678707.2016.1147948 ID - Lum2016 ER - TY - JOUR AU - Lum, S. H. AU - Stepien, K. M. AU - Ghosh, A. AU - Broomfield, A. AU - Church, H. AU - Mercer, J. AU - Jones, S. AU - Wynn, R. PY - 2017 DA - 2017// TI - Long term survival and cardiopulmonary outcome in children with Hurler syndrome after haematopoietic stem cell transplantation JO - J Inherit Metab Dis. VL - 40 UR - https://doi.org/10.1007/s10545-017-0034-6 DO - 10.1007/s10545-017-0034-6 ID - Lum2017 ER - TY - JOUR AU - Grosse, S. D. AU - Lam, W. K. K. AU - Wiggins, L. D. AU - Kemper, A. R. PY - 2017 DA - 2017// TI - Cognitive outcomes and age of detection of severe mucopolysaccharidosis type 1 JO - Genet Med VL - 19 UR - https://doi.org/10.1038/gim.2016.223 DO - 10.1038/gim.2016.223 ID - Grosse2017 ER - TY - JOUR AU - Langereis, E. J. AU - Vlies, N. AU - Wijburg, F. A. PY - 2015 DA - 2015// TI - Diagnosis, classification and treatment of mucopolysaccharidosis type I JO - Expert Opin Orphan Drugs VL - 3 UR - https://doi.org/10.1517/21678707.2015.1016908 DO - 10.1517/21678707.2015.1016908 ID - Langereis2015 ER - TY - JOUR AU - Coletti, H. Y. AU - Aldenhoven, M. AU - Yelin, K. AU - Poe, M. D. PY - 2015 DA - 2015// TI - Long-term functional outcomes of children with Hurler syndrome treated with unrelated umbilical cord blood transplantation JO - JIMD Rep VL - 20 UR - https://doi.org/10.1007/8904_2014_395 DO - 10.1007/8904_2014_395 ID - Coletti2015 ER - TY - JOUR AU - Kiely, B. T. AU - Kohler, J. L. AU - Coletti, H. Y. AU - Poe, M. D. AU - Escolar, M. L. PY - 2017 DA - 2017// TI - Early disease progression of Hurler syndrome JO - Orphanet J Rare Dis VL - 12 UR - https://doi.org/10.1186/s13023-017-0583-7 DO - 10.1186/s13023-017-0583-7 ID - Kiely2017 ER - TY - JOUR AU - Shapiro, E. AU - Guler, O. E. AU - Rudser, K. AU - Delaney, K. PY - 2012 DA - 2012// TI - An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT) JO - Mol Genet Metab VL - 107 UR - https://doi.org/10.1016/j.ymgme.2012.07.016 DO - 10.1016/j.ymgme.2012.07.016 ID - Shapiro2012 ER - TY - JOUR AU - Kuiper, G. A. AU - Hasselt, P. M. AU - Boelens, J. J. AU - Wijburg, F. A. AU - Langereis, E. J. PY - 2017 DA - 2017// TI - Incomplete biomarker response in mucopolysaccharidosis type I after successful hematopoietic cell transplantation JO - Mol Genet Metab VL - 122 UR - https://doi.org/10.1016/j.ymgme.2017.05.009 DO - 10.1016/j.ymgme.2017.05.009 ID - Kuiper2017 ER - TY - JOUR AU - Lund, T. C. AU - Miller, W. P. AU - Liao, A. Y. AU - Tolar, J. PY - 2019 DA - 2019// TI - Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes JO - Sci Rep VL - 9 UR - https://doi.org/10.1038/s41598-019-50595-1 DO - 10.1038/s41598-019-50595-1 ID - Lund2019 ER - TY - JOUR AU - Valayannopoulos, V. AU - Blic, J. AU - Mahlaoui, N. PY - 2010 DA - 2010// TI - Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation JO - Pediatrics VL - 126 UR - https://doi.org/10.1542/peds.2009-2843 DO - 10.1542/peds.2009-2843 ID - Valayannopoulos2010 ER - TY - JOUR AU - Stoop, F. J. AU - Kruyt, M. C. AU - Linden, M. H. AU - Sakkers, R. J. B. PY - 2013 DA - 2013// TI - Prevalence and development of orthopaedic symptoms in the Dutch Hurler patient population after haematopoietic stem cell transplantation JO - JIMD Rep VL - 9 UR - https://doi.org/10.1007/8904_2012_175 DO - 10.1007/8904_2012_175 ID - Stoop2013 ER - TY - JOUR AU - Javed, A. AU - Aslam, T. AU - Jones, S. A. AU - Mercer, J. AU - Tyler, K. AU - Church, H. AU - Ghosh, A. AU - Wynn, R. AU - Sornalingam, K. AU - Ashworth, J. PY - 2018 DA - 2018// TI - The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler) JO - Acta Ophthalmol VL - 96 UR - https://doi.org/10.1111/aos.13627 DO - 10.1111/aos.13627 ID - Javed2018 ER - TY - JOUR AU - Weisstein, J. S. AU - Delgado, E. AU - Steinbach, L. S. AU - Hart, K. AU - Packman, S. PY - 2004 DA - 2004// TI - Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation JO - J Pediatr Orthop. VL - 24 UR - https://doi.org/10.1097/00004694-200401000-00019 DO - 10.1097/00004694-200401000-00019 ID - Weisstein2004 ER - TY - JOUR AU - Schmidt, M. AU - Breyer, S. AU - Löbel, U. AU - Yarar, S. PY - 2016 DA - 2016// TI - Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation JO - Orphanet J Rare Dis VL - 11 UR - https://doi.org/10.1186/s13023-016-0470-7 DO - 10.1186/s13023-016-0470-7 ID - Schmidt2016 ER - TY - JOUR AU - Kuiper, G. A. AU - Langereis, E. J. AU - Breyer, S. AU - Carbone, M. PY - 2019 DA - 2019// TI - Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure JO - Orphanet J Rare Dis VL - 14 UR - https://doi.org/10.1186/s13023-019-0997-5 DO - 10.1186/s13023-019-0997-5 ID - Kuiper2019 ER - TY - BOOK AU - Sempé, M. AU - Pédron, G. AU - Roy-Pernot, M. -. P. PY - 1997 DA - 1997// TI - Auxologie méthode et séquences Théraplix PB - Méditions CY - Paris ID - Sempé1997 ER -