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

المصدر: Liver International. 42:161-172

مصطلحات موضوعية: medicine.medical_specialty, Acetylgalactosamine, Pyrrolidines, givosiran, Urinary system, Acute Hepatic Porprhyria, ALA-synthase-1, givosiran, health-related quality of life, RNAi therapeutics, Attack rate, Placebo, chemistry.chemical_compound, Quality of life, Internal medicine, Porphobilinogen, Humans, Medicine, Adverse effect, ALA-synthase-1, Hepatology, business.industry, Acute Hepatic Porprhyria, RNAi therapeutics, Interim analysis, Porphyrias, Hepatic, health-related quality of life, chemistry, Porphyria, Acute Intermittent, Quality of Life, business, Hemin

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6131b0c2b0931beff9480eb23921ff4eTest
https://doi.org/10.1111/liv.15090Test

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: Husain Attarwala, Valerie A. Clausen, Pushkal Garg, Varun Goel, Verena Karsten, Gabriel J. Robbie, Megan Melch, Akshay Vaishnaw, Bahru A. Habtemariam, John Vest, Marianne T. Sweetser

المصدر: Clinical Pharmacology & Therapeutics. 109:372-382

مصطلحات موضوعية: Adult, Male, Drug, Acetylgalactosamine, media_common.quotation_subject, Pharmacology, 030226 pharmacology & pharmacy, Drug Administration Schedule, N-Acetylgalactosamine, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Asian People, Pharmacokinetics, Humans, Prealbumin, Medicine, Single-Blind Method, Pharmacology (medical), Adverse effect, media_common, Amyloid Neuropathies, Familial, Dose-Response Relationship, Drug, biology, business.industry, Amyloidosis, medicine.disease, Healthy Volunteers, Transthyretin, chemistry, 030220 oncology & carcinogenesis, Pharmacodynamics, biology.protein, RNA, Female, business, Half-Life, Conjugate

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a926fdfa0bbe0b749b1e462a6eaad07dTest
https://doi.org/10.1002/cpt.1974Test

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: Robert J. Fox, Michael Hutchinson, Carlo Pozzilli, Eva Havrdova, K. Selmaj, Ralf Gold, David G. MacManus, Theodore Phillips, James Potts, Minhua Yang, Tarek A. Yousry, Mark Novas, Douglas L. Arnold, Nuwan Kurukulasuriya, Marianne T. Sweetser, Ludwig Kappos, Ray Zhang, Amit Bar-Or, David Miller

المصدر: Multiple Sclerosis (Houndmills, Basingstoke, England)

مصطلحات موضوعية: Oncology, Adult, Male, medicine.medical_specialty, Multiple Sclerosis, Time Factors, Dimethyl Fumarate, Treatment outcome, DEFINE, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Internal medicine, medicine, Humans, 030212 general & internal medicine, ENDORSE, Glatiramer acetate, Expanded Disability Status Scale, Dimethyl fumarate, business.industry, Relapsing–remitting multiple sclerosis, Extension study, Multiple sclerosis, Glatiramer Acetate, medicine.disease, Interim analysis, Magnetic Resonance Imaging, Surgery, delayed-release dimethyl fumarate (DMF), Relapsing-remitting multiple sclerosis, Neurology, Neurology (clinical), Treatment Outcome, chemistry, Delayed-release dimethyl fumarate (DMF), Female, business, Original Research Papers, 030217 neurology & neurosurgery, Immunosuppressive Agents, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c1f78ef34d45e60a75761dd3357f9937Test
http://europepmc.org/articles/PMC5418934Test

المؤلفون: Norman Kim, Marianne T. Sweetser, Patricia Valencia, J. Theodore Phillips, Tim Thullen, Nuwan Kurukulasuriya, Lauren Oliva, Ludwig Kappos, Vissia Viglietta, Robert J. Fox, Amit Bar-Or, Jie Li, Mark Novas, Eva Havrdova, Ralf Gold

المصدر: Neurology and Therapy

مصطلحات موضوعية: medicine.medical_specialty, Neurology, Pharmacology, Multiple sclerosis, chemistry.chemical_compound, Pregnancy, Internal medicine, medicine, Women, Pregnancy outcomes, Delayed-release dimethyl fumarate, Original Research, Dimethyl fumarate, business.industry, medicine.disease, Preclinical, Postmarketing, Clinical trial, Oral agents, chemistry, Neurology (clinical), Safety, business

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1ce364f296eb8857de18f55012f1f98aTest
http://europepmc.org/articles/PMC4685863Test

المؤلفون: Hartmut Schmidt, Jeeyoung Oh, Yesim Parman, Teresa Coelho, Byoung Joon Kim, Akshay Vaishnaw, Ole B. Suhr, Chih-Chao Yang, Scott D. Solomon, Pushkal Garg, Sunita Goyal, Ivailo Tournev, Andrew Strahs, John L. Berk, Pritesh Gandhi, Shahram Attarian, Violaine Planté-Bordeneuve, Alejandra González-Duarte, Arnt V. Kristen, William O'Riordan, Kon Ping Lin, Mitsuharu Ueda, Philip N. Hawkins, Michelle M. Mezei, Jared Gollob, Jihong Chen, David Adams, Saraswathy V. Nochur, Thomas H. Brannagan, Peter J. Dyck, Josep M. Campistol, Giuseppe Vita, Marianne T. Sweetser, Michael Polydefkis, Yoshiki Sekijima, Juan Buades

المساهمون: Universitat de Barcelona

المصدر: Dipòsit Digital de la UB
Universidad de Barcelona
Recercat. Dipósit de la Recerca de Catalunya
instname

مصطلحات موضوعية: Male, Tafamidis, Administration, Intravenous, Adult, Aged, Aged, 80 and over, Amyloid Neuropathies, Familial, Disease Progression, Double-Blind Method, Edema, Female, Gait Disorders, Neurologic, Humans, Infusions, Intravenous, Least-Squares Analysis, Middle Aged, Polyneuropathies, Prealbumin, Quality of Life, RNA, Small Interfering, Severity of Illness Index, Walk Test, RNAi Therapeutics, Medicine (all), 030204 cardiovascular system & hematology, chemistry.chemical_compound, 0302 clinical medicine, Clinical trials, RNA interference, biology, Amyloidosis, General Medicine, 03 medical and health sciences, medicine, business.industry, RNA, medicine.disease, Clinical trial, Transthyretin, chemistry, biology.protein, Cancer research, Amiloïdosi, Amyloid cardiomyopathy, business, human activities, 030217 neurology & neurosurgery, Assaigs clínics

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b94e90139b6836f8b7a776e1f046b770Test
http://hdl.handle.net/2445/138257Test

المؤلفون: Eva Havrdova, Michael Hutchinson, Ralf Gold, Marianne T. Sweetser, Katherine Dawson, Nuwan Kurukulasuriya, Kartik Raghupathi

المصدر: Expert Opinion on Pharmacotherapy. 14:2145-2156

مصطلحات موضوعية: Male, Oncology, medicine.medical_specialty, Disease, Pharmacology, law.invention, chemistry.chemical_compound, Multiple Sclerosis, Relapsing-Remitting, Fumarates, Randomized controlled trial, law, Internal medicine, Humans, Medicine, Pharmacology (medical), Adverse effect, Dimethyl fumarate, business.industry, Multiple sclerosis, Incidence (epidemiology), General Medicine, medicine.disease, Relapsing remitting, chemistry, Expert opinion, Female, Peptides, business, Immunosuppressive Agents

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::feaa7f03888a2d998ac81c6f8b666c5dTest
https://doi.org/10.1517/14656566.2013.826190Test

المؤلفون: Carmen Bozic, Katherine Dawson, Marianne T. Sweetser

المصدر: New England Journal of Medicine. 368:1659-1661

مصطلحات موضوعية: Active ingredient, Dimethyl fumarate, business.industry, Progressive multifocal leukoencephalopathy, Zinc salts, General Medicine, Pharmacology, medicine.disease, chemistry.chemical_compound, chemistry, Fumaric Acid Esters, Compounding, Psoriasis, medicine, Organic chemistry, business

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::6fd1653a0a596f7d7955b87f2c00106cTest
https://doi.org/10.1056/nejmc1300283Test

المؤلفون: J. Theodore Phillips, Mark Novas, Vissia Viglietta, Christian Sindic, Eva Havrdova, Robert J. Fox, Michael Hutchinson, Mariko Kita, Kartik Raghupathi, Katherine Dawson, David Miller, Minhua Yang, Marianne T. Sweetser

المساهمون: UCL - SSS/IONS/CEMO - Pôle Cellulaire et moléculaire

المصدر: The New England journal of medicine, Vol. 367, no. 12, p. 1087-97 (2012)

مصطلحات موضوعية: Male, Adult, medicine.medical_specialty, Pathology, Dimethyl Fumarate, etiology, Aucun, Administration, Oral, drug therapy, pathology, Infections, Placebo, Gastroenterology, law.invention, chemistry.chemical_compound, Multiple Sclerosis, Relapsing-Remitting, Randomized controlled trial, Double-Blind Method, Fumarates, law, Internal medicine, Teriflunomide, medicine, Humans, Glatiramer acetate, Intention-to-treat analysis, Dimethyl fumarate, business.industry, Multiple sclerosis, Brain, Glatiramer Acetate, General Medicine, Middle Aged, medicine.disease, administration & dosage, adverse effects, therapeutic use, Magnetic Resonance Imaging, Intention to Treat Analysis, Clinical trial, chemistry, Female, business, Peptides, Infection, Immunosuppressive Agents, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a543316638f263a695777e838ca797abTest
https://hdl.handle.net/2078.1/124404Test