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1Academic Journal
المؤلفون: Nathan Denton, Andrew E. Mulberg, Monique Molloy, Samantha Charleston, David C. Fajgenbaum, Eric D. Marsh, Paul Howard
المصدر: Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-9 (2022)
مصطلحات موضوعية: Rare diseases, Drug development, Knowledge bases, Registries, Research design, Data management, Medicine
وصف الملف: electronic resource
Relation: https://doaj.org/toc/1750-1172
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2Academic Journal
المؤلفون: Nathan Denton, Andrew E. Mulberg, Monique Molloy, Samantha Charleston, David C. Fajgenbaum, Eric D. Marsh, Paul Howard
المصدر: Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-1 (2023)
مصطلحات موضوعية: Medicine
Relation: https://doi.org/10.1186/s13023-023-02613-9; https://doaj.org/toc/1750-1172; https://doaj.org/article/7624886844a3449395011d8c2f2dc551
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3Academic Journal
المؤلفون: Nathan Denton, Monique Molloy, Samantha Charleston, Craig Lipset, Jonathan Hirsch, Andrew E. Mulberg, Paul Howard, Eric D. Marsh
المصدر: Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-4 (2021)
مصطلحات موضوعية: Rare diseases, Drug development, Knowledge bases, Registries, Research design, Data management, Medicine
وصف الملف: electronic resource
Relation: https://doaj.org/toc/1750-1172
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4Academic Journal
المؤلفون: Melodi Harfouche, Priya S. Kishnani, Eva Krusinska, Jamie Gault, Sheela Sitaraman, Amanda Sowinski, Irina Katz, Stephanie Austin, Margi Goldstein, Andrew E. Mulberg
المصدر: Journal of Patient-Reported Outcomes, Vol 4, Iss 1, Pp 1-11 (2020)
مصطلحات موضوعية: PROMIS, Pompe disease, Patient-reported outcomes (PROs), Clinical research, Drug development, Public aspects of medicine, RA1-1270
وصف الملف: electronic resource
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5Academic Journal
المؤلفون: Andrew E. Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, David Jacoby, Franklin K. Johnson, Qing Liu, Deborah Marsden, Scott McGoohan, Robert Nelson, Nita Patel, Klaus Romero, Vikram Sinha, Sheela Sitaraman, John Spaltro, Vivian Kessler
المصدر: Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-10 (2019)
مصطلحات موضوعية: Medicine
وصف الملف: electronic resource
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6Academic Journal
المؤلفون: Andrew E. Mulberg, MD, FAAP, Timothy Cripps, MSc
المصدر: Current Therapeutic Research, Vol 90, Iss , Pp 109-112 (2019)
مصطلحات موضوعية: Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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7
المؤلفون: Timothy Cripps, Andrew E. Mulberg
المصدر: Pediatric Inflammatory Bowel Disease ISBN: 9783031147432
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8
المؤلفون: Andrew E. Mulberg, Alan L. Shields, Vivian Kessler, Nina Skuban, Jay A. Barth, Fiona Taylor, Roger E. Lamoureux
المصدر: Quality of Life Research
مصطلحات موضوعية: Diarrhea, Gastrointestinal signs and symptoms, medicine.medical_specialty, Pediatrics, Article, 03 medical and health sciences, 0302 clinical medicine, Bloating, Content validity, Humans, Medicine, Patient Reported Outcome Measures, 030212 general & internal medicine, Fabry disease, 0303 health sciences, Patient-reported outcomes, business.industry, Public health, Debriefing, 030305 genetics & heredity, Public Health, Environmental and Occupational Health, Models, Theoretical, medicine.disease, Clinical trial, Quality of Life, Patient-reported outcome, medicine.symptom, business, FABPRO-GI
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9
المؤلفون: Andrew E. Mulberg, Laurie S. Conklin, Nicholas M. Croft, Anushree Parikh, Athos Bousvaros, Edwin DeZoeten, Marla Dubinsky, Francisco Javier Martin Carpi, Hankje Escher, Richard Vesely, Cecile Ollivier, Wallace Crandall, Collin Hovinga
المساهمون: Pediatrics
المصدر: Gastroenterology, 163(1), 77-83. W.B. Saunders
مصطلحات موضوعية: Adult, Drug Development, Hepatology, United States Food and Drug Administration, Age Factors, Gastroenterology, Humans, Child, United States
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10
المؤلفون: Drucy Borowitz, Nell Aronoff, Linda C. Cummings, Asim Maqbool, Andrew E. Mulberg
المصدر: Pancreas. 51(4)
مصطلحات موضوعية: Endocrinology, Coal, Hepatology, Cystic Fibrosis, Endocrinology, Diabetes and Metabolism, Internal Medicine, Humans, Reproducibility of Results, Enzyme Replacement Therapy, Exocrine Pancreatic Insufficiency
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11
المؤلفون: Lawrence Goldkind, Janice M. Soreth, Andrew E. Mulberg
المصدر: Gastroenterology.
مصطلحات موضوعية: Hepatology, Gastroenterology
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12
المؤلفون: Lawrence, Goldkind, Andrew E, Mulberg, Janice, Soreth
المصدر: Gastroenterology. 164:496
مصطلحات موضوعية: Hepatology, Gastroenterology
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13
المؤلفون: Donna Griebel, Insook Kim, Robert M. Nelson, Jeremiah D. Momper, M. Tobias Heinrichs, Issam Zineh, Yeruk Mulugeta, Nitin Mehrotra, Shaun Kumar, Gregory H. Reaman, Vikram Sinha, Hari Cheryl Sachs, Narayana Garimella, Gilbert J. Burckart, Lynne Yao, Kevin Krudys, Andrew E. Mulberg
المصدر: The Journal of Clinical Pharmacology. 60:775-784
مصطلحات موضوعية: Male, Pediatrics, 030226 pharmacology & pharmacy, 0302 clinical medicine, Neurokinin-1 Receptor Antagonists, Drug Dosage Calculations, Pharmacology (medical), Child, Aprepitant, Aged, 80 and over, Clinical Trials as Topic, Palonosetron, Nausea, Middle Aged, Ondansetron, Treatment Outcome, Child, Preschool, Data Interpretation, Statistical, 030220 oncology & carcinogenesis, Female, medicine.symptom, medicine.drug, Adult, medicine.medical_specialty, Adolescent, Vomiting, medicine.drug_class, Antineoplastic Agents, Granisetron, Young Adult, 03 medical and health sciences, medicine, Humans, Antiemetic, Adverse effect, Aged, Pharmacology, Dose-Response Relationship, Drug, United States Food and Drug Administration, business.industry, Infant, United States, Clinical trial, Antiemetics, business, Chemotherapy-induced nausea and vomiting
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14Expediting Drug Development for Pediatric Inflammatory Bowel Disease: A Discussion With Stakeholders
المؤلفون: Richard B. Colletti, Athos Bousvaros, Shehzad Ahmed Saeed, Andrew E. Mulberg, Eric Zuckerman, Harland S. Winter, Anne M. Robinson, Perdita Taylor-Zapata, Jeffrey S. Hyams, Joachim Musaus, Kerry Jo Lee, Joel R. Rosh
المصدر: Journal of Pediatric Gastroenterology & Nutrition. 66:95-99
مصطلحات موضوعية: medicine.medical_specialty, Expediting, business.industry, Gastroenterology, Pharmacology, medicine.disease, Inflammatory bowel disease, 03 medical and health sciences, 0302 clinical medicine, Drug development, 030225 pediatrics, Pediatrics, Perinatology and Child Health, medicine, 030211 gastroenterology & hepatology, Intensive care medicine, business
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15
المؤلفون: Jian Wang, Farrokh Sohrabi, Sue-Chih Lee, Andrew E. Mulberg, Dennis Bashaw, Vikram Sinha, Hae-Young Ahn, Insook Kim, Ruyi He, Nitin Mehrotra, Donna Griebel
المصدر: Journal of Pediatric Gastroenterology & Nutrition. 63:412-416
مصطلحات موضوعية: Male, Quinuclidines, Pediatrics, medicine.medical_specialty, Adolescent, Vomiting, Nausea, Antineoplastic Agents, 030226 pharmacology & pharmacy, Drug Administration Schedule, 03 medical and health sciences, 0302 clinical medicine, Pharmacokinetics, medicine, Humans, Child, Infusions, Intravenous, Response rate (survey), Dose-Response Relationship, Drug, business.industry, Palonosetron, Gastroenterology, Area under the curve, Infant, Isoquinolines, Clinical trial, Logistic Models, Treatment Outcome, Area Under Curve, Child, Preschool, 030220 oncology & carcinogenesis, Pediatrics, Perinatology and Child Health, Antiemetics, Female, medicine.symptom, business, medicine.drug, Chemotherapy-induced nausea and vomiting
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16
المؤلفون: Elektra J. Papadopoulos, Benjamin Lebwohl, Juli Tomaino, Sonia S. Kupfer, Daniel A. Leffler, Andrew E. Mulberg, Kevin Bugin, Julia Tait Lathrop, Jessica J. Lee, Sheila E. Crowe, Donna Griebel
المصدر: Gastroenterology. 151:407-411
مصطلحات موضوعية: Health related quality of life, medicine.medical_specialty, Hepatology, business.industry, Gastroenterology, MEDLINE, Disease, Food and drug administration, 03 medical and health sciences, 0302 clinical medicine, 030220 oncology & carcinogenesis, Internal medicine, Drug approval, Medicine, 030211 gastroenterology & hepatology, Pharmaceutical sciences, business
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17
المؤلفون: Marla Dubinsky, Jeremiah D. Momper, Nitin Mehrotra, Karim Azer, Laurie S. Conklin, Andrew E. Mulberg, Dan Turner, William A. Faubion
المصدر: Quantitative Pharmacology and Individualized Therapy Strategies in Development of Therapeutic Proteins for Immune-Mediated Inflammatory Diseases ISBN: 9781119289234
Quantitative Pharmacology and Individualized Therapy Strategies in Development of Therapeutic Proteins for Immune-Mediated Inflammatory Diseasesمصطلحات موضوعية: medicine.medical_specialty, business.industry, Quantitative pharmacology, Adalimumab, Medicine, business, Intensive care medicine, medicine.disease, Inflammatory bowel disease, Infliximab, medicine.drug, Pediatric population
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18
المؤلفون: Severine Vermeire, Bill Sandborn, Bruce E. Sands, Jean-Frederic Colombel, Remo Panaccione, Laurent Peyrin-Biroulet, Walter Reinisch, Klaus Gottlieb, David T. Rubin, Andrew E. Mulberg, Silvio Danese, Julián Panés, Stefan Schreiber
المساهمون: Reinisch, W, Gottlieb, K, Colombel, Jf, Danese, S, Panaccione, R, Panes, J, Peyrin-Biroulet, L, Rubin, D, Sands, Be, Schreiber, S, Vermeire, S, Mulberg, A, Sandborn, B
مصطلحات موضوعية: medicine.medical_specialty, Concordance, Harmonization, Guidelines as Topic, Subject matter, 03 medical and health sciences, 0302 clinical medicine, Government Agencies, Drug Development, medicine, Humans, Medical physics, Regulatory science, European Union, Patient Reported Outcome Measures, Intestinal Mucosa, health care economics and organizations, Clinical Trials as Topic, Hepatology, business.industry, United States Food and Drug Administration, Gastroenterology, Guideline, medicine.disease, Ulcerative colitis, United States, Treatment Outcome, Drug development, 030220 oncology & carcinogenesis, 030211 gastroenterology & hepatology, Patient-reported outcome, Colitis, Ulcerative, business
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19
المؤلفون: K.T. Park, Laurie S. Conklin, Athos Bousvaros, Dale Lee, Caren Heller, Anthony R. Otley, David Ziring, Andrew B. Grossman, Andrew E. Mulberg, Alandra Weaver, Subra Kugathasan, Jeffrey S. Hyams, Joel R. Rosh, Joseph A. Picoraro, Michael D. Kappelman, Lee A. Denson
المصدر: Inflamm Bowel Dis
مصطلحات موضوعية: medicine.medical_specialty, Translational research, Disease, Inflammatory bowel disease, 03 medical and health sciences, 0302 clinical medicine, Crohn Disease, 030225 pediatrics, medicine, Immunology and Allergy, Humans, Colitis, Disease management (health), Leading off, Intensive care medicine, Child, Irritable bowel syndrome, Crohn's disease, business.industry, Tumor Necrosis Factor-alpha, Gastroenterology, Antibodies, Monoclonal, Disease Management, medicine.disease, Prognosis, Ulcerative colitis, digestive system diseases, Practice Guidelines as Topic, 030211 gastroenterology & hepatology, Colitis, Ulcerative, business
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20
المؤلفون: Fred Holdbrook, J. Charles Jennette, Jay A. Barth, Jeffrey P. Castelli, Robert B. Colvin, Kathleen Nicholls, Suma P. Shankar, Laura Barisoni, Daniel G. Bichet, Derralynn Hughes, Andrew E. Mulberg, Nina Skuban, Raphael Schiffmann, Ana Jovanovic, Ulla Feldt-Rasmussen, Roberto Giugliani
المصدر: Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-7 (2018)
Repositório Institucional da UFRGS
Universidade Federal do Rio Grande do Sul (UFRGS)
instacron:UFRGS
Schiffmann, R, Bichet, D G, Jovanovic, A, Hughes, D A, Giugliani, R, Feldt-Rasmussen, U, Shankar, S P, Barisoni, L, Colvin, R B, Jennette, J C, Holdbrook, F, Mulberg, A, Castelli, J P, Skuban, N, Barth, J A & Nicholls, K 2018, ' Migalastat improves diarrhea in patients with Fabry disease : clinical-biomarker correlations from the phase 3 FACETS trial ', Orphanet Journal of Rare Diseases, vol. 13, 68 . https://doi.org/10.1186/s13023-018-0813-7
Orphanet Journal of Rare Diseases
Orphanet journal of rare diseases, vol 13, iss 1مصطلحات موضوعية: Male, 0301 basic medicine, GSRS, lcsh:Medicine, Neurodegenerative, Kidney, Biomarkers/metabolism, Gastroenterology, Doença de Fabry, Globotriaosylceramide, chemistry.chemical_compound, 0302 clinical medicine, Migalastat, Lyso-Gb3, Pharmacological chaperone, Pharmacology (medical), Diferença mínima clinicamente importante, Diarréia, 1-Deoxynojirimycin/analogs & derivatives, Genetics (clinical), Genetics & Heredity, Trihexosylceramides, Minimal clinically important difference, General Medicine, Middle Aged, Fabry Disease/drug therapy, Diarrhea, 6.1 Pharmaceuticals, Female, medicine.symptom, Adult, Diarrhea/drug therapy, Gastrointestinal, medicine.medical_specialty, 1-Deoxynojirimycin, Adolescent, Mutation/genetics, Clinical Trials and Supportive Activities, Placebo, Young Adult, 03 medical and health sciences, Rare Diseases, Clinical Research, Internal medicine, medicine, Humans, In patient, Aged, Fabry disease, Other Medical and Health Sciences, Surrogate endpoint, business.industry, Research, lcsh:R, Evaluation of treatments and therapeutic interventions, medicine.disease, Brain Disorders, Kidney/metabolism, Emerging Infectious Diseases, 030104 developmental biology, chemistry, Mutation, Amenable mutation, Digestive Diseases, business, Biomarkers, 030217 neurology & neurosurgery
وصف الملف: application/pdf