المؤلفون: Pushkal Garg, William O'Riordan, Sally A. Hulton, John C. Lieske, Jaap W. Groothoff, Eva Simkova, Hadas Shasha-Lavsky, Sander F. Garrelfs, Akshay Vaishnaw, Gesa Schalk, John M. Gansner, Kristin Meliambro, Pierre Cochat, Daniella Magen, Daniel Guido Fuster, Marianne T. Sweetser, William van’t Hoff, Jiandong Lu, Michael J. Koren, Yaacov Frishberg, Jeffrey M. Saland, Shabbir H. Moochhala, Georges Deschênes, Tracy L. McGregor, David J. Sas

المساهمون: Graduate School, APH - Methodology, APH - Quality of Care, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Paediatric Nephrology, ARD - Amsterdam Reproduction and Development

المصدر: New England journal of medicine, 384(13), 1216-1226. Massachussetts Medical Society

مصطلحات موضوعية: medicine.medical_specialty, Renal function, Disease, 030204 cardiovascular system & hematology, Gastroenterology, Oxalate, RNAi Therapeutics, Primary hyperoxaluria, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Internal medicine, medicine, 030212 general & internal medicine, 610 Medicine & health, Kidney, urogenital system, business.industry, General Medicine, medicine.disease, medicine.anatomical_structure, chemistry, 570 Life sciences, biology, Kidney stones, Nephrocalcinosis, business

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c371005175d1307f379da84b8ad2b16fTest
http://www.scopus.com/inward/record.url?scp=85103565761&partnerID=8YFLogxKTest

المؤلفون: Paolo Ventura, Eliane Sardh, Nicola Longo, Manisha Balwani, Jorge Plutzky, Laurent Gouya, John Phillips, Sean Rhyee, Mary-Jean Fanelli, Marianne T. Sweetser, Petro E. Petrides

مصطلحات موضوعية: Clinical Trials as Topic, Acute Hepatic Porphyria (AHP), Hepatology, givosiran, Hyperhomocysteinemia, Gastroenterology, Cystathionine beta-Synthase, Pyridoxine, Heme, homocysteine, Acute Intermittent Porprhyria (AIP), small interfering RNA, Vitamin B 6, Porphyrias, Hepatic, Folic Acid, Methionine, Humans, RNA, Small Interfering, Acute Hepatic Porphyria (AHP), Acute Intermittent Porprhyria (AIP), givosiran, homocysteine,hyperhomocysteinemia, small interfering RNA, hyperhomocysteinemia, Homocysteine, Sulfur

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f5f3ca29086dc98d640e06f29a98851fTest
https://hdl.handle.net/11380/1286364Test

المؤلفون: Manish Thapar, Herbert L. Bonkovsky, Susana Monroy, Gayle Ross, Encarna Guillén-Navarro, Maria Domenica Cappellini, Anna-Elisabeth Minder, Shangbin Liu, Marianne T. Sweetser, David J. Kuter

المصدر: Gastroenterology. 162:S-80

مصطلحات موضوعية: Hepatology, Gastroenterology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::3d90a07dcc2976494309ae01c3ab35caTest
https://doi.org/10.1016/s0016-5085Test(22)60206-3

المؤلفون: Laurent Gouya, D. Montgomery Bissell, David J. Kuter, Marianne T. Sweetser, Herbert L. Bonkovsky, Paolo Ventura, Paula Aguilera Peiró, Eliane Sardh, Karl E. Anderson, Jeeyoung Oh, Penelope E. Stein, Zhaowei Hua, John J. Ko, Susana Monroy

المصدر: American Journal of Gastroenterology. 116:S545-S546

مصطلحات موضوعية: Acute hepatic porphyria, medicine.medical_specialty, Hepatology, Randomized controlled trial, business.industry, law, Internal medicine, Gastroenterology, Medicine, In patient, business, Interim analysis, law.invention

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::7211b2ec87cf0d20182a2ccd4827c613Test
https://doi.org/10.14309/01.ajg.0000778212.30789.aeTest

المؤلفون: Matthew T. White, Michael Polydefkis, Marianne T. Sweetser, Angela Dispenzieri, John L. Berk, Jing Jing Wang, Julian D. Gillmore, Mathew S. Maurer, Alejandra González-Duarte, Seth Arum, Yoshiki Sekijima

المصدر: European Heart Journal. 41

مصطلحات موضوعية: medicine.medical_specialty, biology, business.industry, MedDRA, Mortality rate, Amyloidosis, Cardiomyopathy, Atrial fibrillation, medicine.disease, Gastroenterology, Transthyretin, Internal medicine, Cohort, medicine, biology.protein, In patient, Adverse effect, Cardiology and Cardiovascular Medicine, business, Polyneuropathy

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7b0521a23e3a0e4f52208d9be9a8155Test
https://doi.org/10.1093/ehjci/ehaa946.2129Test

المؤلفون: Marianne T. Sweetser, Márcia Waddington-Cruz, Josep M. Campistol, Hartmut Schmidt, John L. Berk, Jing Jing Wang, Jared Gollob, David H. Adams, Teresa Coelho, Juan Buades, Michael Polydefkis, Jihong Chen, Ole B. Suhr, Isabel Conceição

المساهمون: Repositório da Universidade de Lisboa

المصدر: Orphanet Journal of Rare Diseases
Repositório Científico de Acesso Aberto de Portugal
Repositório Científico de Acesso Aberto de Portugal (RCAAP)
instacron:RCAAP
Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-10 (2020)

مصطلحات موضوعية: Tafamidis, medicine.medical_specialty, Neurologi, Cardiomyopathy, lcsh:Medicine, 030204 cardiovascular system & hematology, Gastroenterology, 03 medical and health sciences, chemistry.chemical_compound, RNA interference, 0302 clinical medicine, NA interference, Polyneuropathy, Internal medicine, medicine, Humans, Prealbumin, ATTR amyloidosis, Pharmacology (medical), RNA, Small Interfering, Adverse effect, Genetics (clinical), Amyloid Neuropathies, Familial, biology, business.industry, Research, Amyloidosis, lcsh:R, General Medicine, medicine.disease, Discontinuation, Transthyretin, Neurology, chemistry, Concomitant, Quality of Life, Patisiran, biology.protein, business, 030217 neurology & neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4ab76833c3c740a5a556579909bdfbdaTest
https://doi.org/10.1186/s13023-020-01399-4Test

المؤلفون: Herbert L. Bonkovsky, Ming-Jen Lee, David Cassiman, Aneta Ivanova, Susana Monroy, Encarna Guillen-Navarro, Marianne T. Sweetser, Zhaowei Hua, Facg, Manish Thapar, Raili M. Kauppinen

المصدر: American Journal of Gastroenterology. 116:S546-S546

مصطلحات موضوعية: Acute hepatic porphyria, Pediatrics, medicine.medical_specialty, Hepatology, business.industry, Gastroenterology, medicine, business, Natural history study, Term (time)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::b50c061b5e4f4a12628fbc3267855a2bTest
https://doi.org/10.14309/01.ajg.0000778216.45955.7fTest

المؤلفون: Paolo Ventura, Bruce Wang, Manisha Balwani, Marianne T. Sweetser, Manish Thapar, Zhaowei Hua, Kei-ichiro Takase, Ilja Kubisch, David Cassiman

المصدر: American Journal of Gastroenterology. 116:S504-S504

مصطلحات موضوعية: Acute hepatic porphyria, medicine.medical_specialty, Hepatology, business.industry, Internal medicine, Gastroenterology, medicine, In patient, business, Disease burden

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::4edf2caa72042a5c57b9479b23d941c8Test
https://doi.org/10.14309/01.ajg.0000777784.56221.d2Test

المؤلفون: Xiaojun You, James G. Krueger, Firas Hougeir, Adam J. Friedman, Nisha Lucas, Marianne T. Sweetser, Jacob Elkins, Steven J. Greenberg, Wanda Castro-Borrero, Leon H Kircik, Peter McCroskery

المصدر: Advances in Therapy

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Daclizumab, MedDRA, Phases of clinical research, Dermatology, Antibodies, Monoclonal, Humanized, Severity of Illness Index, Skin Diseases, Gastroenterology, 030207 dermatology & venereal diseases, 03 medical and health sciences, Multiple Sclerosis, Relapsing-Remitting, 0302 clinical medicine, Double-Blind Method, Internal medicine, Severity of illness, medicine, Humans, Pharmacology (medical), Adverse effect, Daclizumab high-yield process, Original Research, Cutaneous events, Medicine(all), business.industry, Incidence, Multiple sclerosis, Interferon beta-1a, General Medicine, Middle Aged, Interferon beta, medicine.disease, Rash, Treatment Outcome, Neurology, Immunoglobulin G, Immunology, Relapsing-remitting multiple sclerosis, Female, Safety, medicine.symptom, business, 030217 neurology & neurosurgery, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a38992de9f4f237336503be280f49005Test
https://doi.org/10.1007/s12325-016-0353-2Test

المؤلفون: Marianne T. Sweetser, Ludwig Kappos, Steven Greenberg, Michael Kaufman, Alexey Boyko, Katherine Riester, Douglas L. Arnold, Gilmore O'neill, Heinz Wiendl, Krzysztof Selmaj, Jacob Elkins, John W. Rose, Eva Havrdova

المصدر: New England Journal of Medicine. 373:1418-1428

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Daclizumab, medicine.drug_class, Phases of clinical research, Kaplan-Meier Estimate, Antibodies, Monoclonal, Humanized, Monoclonal antibody, Gastroenterology, Multiple Sclerosis, Relapsing-Remitting, Adjuvants, Immunologic, Double-Blind Method, Interferon, Internal medicine, Humans, Medicine, IL-2 receptor, business.industry, Multiple sclerosis, Interferon beta-1a, Interferon-beta, General Medicine, Middle Aged, medicine.disease, Endocrinology, Immunoglobulin G, Monoclonal, Disease Progression, Female, business, Immunosuppressive Agents, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::063f9f5cd3c15f0e97211149013b459dTest
https://doi.org/10.1056/nejmoa1501481Test