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

URL الوصول: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942017000100803

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

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

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

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

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

المؤلفون: Berger, Kenneth I.^{Aff13, Aff14}, Burton, Barbara K.^Aff15, Lewis, Gregory D.^Aff16, Tarnopolsky, Mark^Aff17, Harmatz, Paul R.^Aff18, Mitchell, John J.^Aff19, Muschol, Nicole^Aff20, Jones, Simon A.^Aff21, Sutton, V. Reid^Aff22, Pastores, Gregory M.^{Aff13, Aff23}, Lau, Heather^Aff13, Sparkes, Rebecca^Aff24, Shaywitz, Adam J.^Aff25

المساهمون: Baumgartner, Matthias, Series Editor^{Aff1, Aff8}, Patterson, Marc, Series Editor^{Aff2, Aff9}, Rahman, Shamima, Series Editor^{Aff3, Aff10}, Peters, Verena, Series Editor^{Aff4, Aff12}, Morava, Eva, Editor-in-Chief^{Aff5, Aff7}, Zschocke, Johannes, Series Editor^{Aff6, Aff11}

المصدر: JIMD Reports, Volume 42. 42:9-17

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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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