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1دورية أكاديمية
المؤلفون: Treadwell, Marsha, Harmatz, Paul R., Burton, Barbara K., Mitchell, John J., Muschol, Nicole, Jones, Simon A., Pastores, Gregory M., Lau, Heather A., Sparkes, Rebecca, Sutton, V. Reid, Meesen, Bianca, Haller, Christine A., Shaywitz, Adam J., Gold, Jeffrey I.
المصدر: Journal of Inborn Errors of Metabolism and Screening. January 2017 5
مصطلحات موضوعية: Morquio A syndrome, chronic pain, ysosomal storage diseases, galactosamine-6-sulfatase, enzyme replacement therapy, elosulfase alfa
وصف الملف: text/html
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2دورية أكاديمية
المؤلفون: Muschol, Nicole, Koehn, Anja, von Cossel, Katharina, Okur, Ilyas, Ezgu, Fatih, Harmatz, Paul, de Castro Lopez, Maria Jose, Couce, Maria Luz, Lin, Shuan-Pei, Batzios, Spyros, Cleary, Maureen, Solano, Martha, Nestrasil, Igor, Kaufman, Brian D, Shaywitz, Adam J, Maricich, Stephen M, Kuca, Bernice, Kovalchin, Joseph, Zanelli, Eric H
المصدر: Journal of Clinical Investigation. 133(2)
مصطلحات موضوعية: Rare Diseases, Brain Disorders, Orphan Drug, Mucopolysaccharidoses (MPS), Neurosciences, Evaluation of treatments and therapeutic interventions, Development of treatments and therapeutic interventions, 5.1 Pharmaceuticals, 6.1 Pharmaceuticals, Humans, Mucopolysaccharidosis III, Heparitin Sulfate, Brain, Liver, Spleen, Neurological disorders, Neuroscience, Medical and Health Sciences, Immunology
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/3f68v4xr
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3دورية أكاديمية
المؤلفون: Berger, Kenneth I, Burton, Barbara K, Lewis, Gregory D, Tarnopolsky, Mark, Harmatz, Paul R, Mitchell, John J, Muschol, Nicole, Jones, Simon A, Sutton, V Reid, Pastores, Gregory M, Lau, Heather, Sparkes, Rebecca, Shaywitz, Adam J
مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Clinical Trials and Supportive Activities, Lung, Clinical Research, Evaluation of treatments and therapeutic interventions, 6.7 Physical, 6.1 Pharmaceuticals, Cardiopulmonary exercise test, Elosulfase alfa, Endurance, Enzyme replacement therapy, Exercise capacity, Mucopolysaccharidosis IVA, Clinical sciences
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/3d9094jt
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4دورية أكاديمية
المؤلفون: Jones, Simon A, Bialer, Martin, Parini, Rossella, Martin, Ken, Wang, Hui, Yang, Ke, Shaywitz, Adam J, Harmatz, Paul
المصدر: Pediatric Research. 78(6)
مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Clinical Trials and Supportive Activities, Clinical Research, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Age Factors, Biomarkers, Body Height, Child Development, Child, Preschool, Chondroitinsulfatases, Drug Administration Schedule, Early Medical Intervention, Enzyme Replacement Therapy, Europe, Female, Humans, Infant, Infusions, Intravenous, Keratan Sulfate, Male, Mucopolysaccharidosis IV, Recombinant Proteins, Time Factors, Treatment Outcome, United Kingdom, Paediatrics and Reproductive Medicine, Public Health and Health Services, Pediatrics, Paediatrics
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/1wh573m2
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5دورية أكاديمية
المؤلفون: Burton, Barbara K, Berger, Kenneth I, Lewis, Gregory D, Tarnopolsky, Mark, Treadwell, Marsha, Mitchell, John J, Muschol, Nicole, Jones, Simon A, Sutton, V Reid, Pastores, Gregory M, Lau, Heather, Sparkes, Rebecca, Genter, Fred, Shaywitz, Adam J, Harmatz, Paul
المصدر: American Journal of Medical Genetics Part A. 167(10)
مصطلحات موضوعية: Pharmacology and Pharmaceutical Sciences, Biomedical and Clinical Sciences, Clinical Sciences, Clinical Trials and Supportive Activities, Pain Research, Chronic Pain, Cardiovascular, Clinical Research, Evaluation of treatments and therapeutic interventions, 6.7 Physical, 6.1 Pharmaceuticals, Musculoskeletal, Adolescent, Adult, Child, Chondroitinsulfatases, Double-Blind Method, Drug Administration Schedule, Enzyme Replacement Therapy, Exercise Test, Female, Heart Function Tests, Humans, Keratan Sulfate, Male, Mucopolysaccharidosis IV, Muscle Strength, Patient Safety, Pilot Projects, Recombinant Proteins, Respiratory Function Tests, Treatment Outcome, Walking, mucopolysaccharidosis IV, cardiopulmonary exercise test, safety, respiratory function tests, muscle strength, physical endurance, GALNS protein, human [supplementary concept], enzyme replacement therapy, Genetics, Clinical sciences
وصف الملف: application/pdf
URL الوصول: https://escholarship.org/uc/item/119351pp
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6دورية أكاديمية
المؤلفون: Grover, Anita, Crippen-Harmon, Danielle, Nave, Lacey, Vincelette, Jon, Wait, Jill C. M., Melton, Andrew C., Lawrence, Roger, Brown, Jillian R., Webster, Katherine A., Yip, Bryan K., Baridon, Brian, Vitelli, Catherine, Rigney, Sara, Christianson, Terri M., Tiger, Pascale M. N., Lo, Melanie J., Holtzinger, John, Shaywitz, Adam J., Crawford, Brett E., Fitzpatrick, Paul A., LeBowitz, Jonathan H., Bullens, Sherry, Aoyagi-Scharber, Mika, Bunting, Stuart, O’Neill, Charles A.Aff1, Pinkstaff, Jason, Bagri, Anil
المصدر: Drug Delivery and Translational Research: An Official Journal of the Controlled Release Society. 10(2):425-439
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7كتاب إلكتروني
المؤلفون: Berger, Kenneth I.Aff13, Aff14, Burton, Barbara K.Aff15, Lewis, Gregory D.Aff16, Tarnopolsky, MarkAff17, Harmatz, Paul R.Aff18, Mitchell, John J.Aff19, Muschol, NicoleAff20, Jones, Simon A.Aff21, Sutton, V. ReidAff22, Pastores, Gregory M.Aff13, Aff23, Lau, HeatherAff13, Sparkes, RebeccaAff24, Shaywitz, Adam J.Aff25
المساهمون: Baumgartner, Matthias, Series EditorAff1, Aff8, Patterson, Marc, Series EditorAff2, Aff9, Rahman, Shamima, Series EditorAff3, Aff10, Peters, Verena, Series EditorAff4, Aff12, Morava, Eva, Editor-in-ChiefAff5, Aff7, Zschocke, Johannes, Series EditorAff6, Aff11
المصدر: JIMD Reports, Volume 42. 42:9-17
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8دورية أكاديمية
المؤلفون: West, Anne E., Chen, Wen G., Dalva, Matthew B., Dolmetsch, Ricardo E., Kornhauser, Jon M., Shaywitz, Adam J., Takasu, Mari A., Tao, Xu, Greenberg, Michael E.
المصدر: Proceedings of the National Academy of Sciences of the United States of America, 2001 Sep . 98(20), 11024-11031.
URL الوصول: https://www.jstor.org/stable/3056662
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9دورية أكاديمية
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10دورية أكاديمية
المؤلفون: Schweighardt, Becky, Tompkins, Troy, Lau, Kelly, Jesaitis, Lynne, Qi, Yulan, Musson, Donald G., Farmer, Pamela, Haller, Christine, Shaywitz, Adam J., Yang, Ke, O’Neill, Charles A.
المصدر: In Clinical Therapeutics 1 May 2015 37(5):1012-1021