المؤلفون: 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

المصدر: Orphanet Journal of Rare Diseases. 14(1)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Mucopolysaccharidoses (MPS), Clinical Research, Rare Diseases, 7.3 Management and decision making, Management of diseases and conditions, Chondroitinsulfatases, Enzyme Replacement Therapy, Female, Humans, Hypercapnia, Male, Mucopolysaccharidosis IV, Morquio a syndrome, Mucopolysaccharidosis, MPS IVA, Management guidelines, Elosulfase alfa, VIMIZIM, Enzyme replacement therapy, ERT, Haematopoietic stem cell transplantation, HSCT, Surgery, Anaesthetics, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs, Other Medical and Health Sciences, Genetics & Heredity, Genetics, Clinical sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/6j0140dz

المؤلفون: 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

المصدر: Orphanet Journal of Rare Diseases. 14(1)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Rare Diseases, Orphan Drug, Mucopolysaccharidoses (MPS), 7.3 Management and decision making, Management of diseases and conditions, Activities of Daily Living, Consensus, Disease Management, Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplantation, Humans, Mucopolysaccharidoses, Mucopolysaccharidosis VI, N-Acetylgalactosamine-4-Sulfatase, Quality of Life, Recombinant Proteins, Maroteaux-Lamy syndrome, Mucopolysaccharidosis, MPS VI, Management guidelines, Galsulfase, Enzyme replacement therapy, ERT, Haematopoietic stem cell transplantation, HSCT, Surgery, Anaesthetics, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs, Other Medical and Health Sciences, Genetics & Heredity, Genetics, Clinical sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/5pc012vk

المؤلفون: Horgan, Claire, Jones, Simon A., Bigger, Brian W., Wynn, Robert

المصدر: International Journal of Molecular Sciences; May2022, Vol. 23 Issue 9, p4854-4854, 18p

مصطلحات موضوعية: STEM cell treatment, HEMATOPOIETIC stem cells, MUCOPOLYSACCHARIDOSIS, LYSOSOMAL storage diseases, STEM cell transplantation, LYSOSOMES

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المؤلفون: Amy Sutton, Heather J. Church, Carr Trevor, Wynn Robert, Sonya Zaman, Wraith Ed, Grainger John

المصدر: Blood. 106:2058-2058

مصطلحات موضوعية: medicine.medical_specialty, Allogeneic transplantation, business.industry, Mucopolysaccharidosis, Urinary system, Immunology, Cell Biology, Hematology, medicine.disease, Biochemistry, Surgery, Transplantation, Exact test, Mucopolysaccharidosis type I, surgical procedures, operative, Graft-versus-host disease, Mucopolysaccharidosis I, medicine, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::f3e7068eca448f7072659e0e7fcdb554
https://doi.org/10.1182/blood.v106.11.2058.2058

المؤلفون: Langford-Smith, Alex, Langford-Smith, Kia J., Jones, Simon A., Wynn, Robert F., Wraith, J. E., Wilkinson, Fiona L., Bigger, Brian W.

المصدر: PLoS ONE; 2011, Vol. 6 Issue 10, p1-14, 14p

مصطلحات موضوعية: MUCOPOLYSACCHARIDOSIS, NEURODEGENERATION, LYSOSOMAL storage diseases, CIRCADIAN rhythms

المؤلفون: de Ru, Minke H, Boelens, Jaap J, Das, Anibh M, Jones, Simon A, van der Lee, Johanna H, Mahlaoui, Nizar, Mengel, Eugen, Offringa, Martin, O'Meara, Anne, Parini, Rossella, Rovelli, Attilio, Sykora, Karl-Walter, Valayannopoulos, Vassili, Vellodi, Ashok, Wynn, Robert F, Wijburg, Frits A

المصدر: Orphanet Journal of Rare Diseases; 2011, Vol. 6 Issue 1, p55-63, 9p

مصطلحات موضوعية: MUCOPOLYSACCHARIDOSIS treatment, SPECIALISTS, ENZYMES, HEMATOPOIETIC stem cell transplantation, MUCOPOLYSACCHARIDOSIS