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

المصدر: Orphanet Journal of Rare Diseases, vol 17, iss 1

مصطلحات موضوعية: Acute Porphyrias, Acute hepatic porphyria, Givosiran, Disease burden, Porphyria attack, Chronic symptoms, Quality of life, Porphobilinogen, Neurodegenerative, Research & Experimental Medicine, ACUTE INTERMITTENT PORPHYRIA, Hepatic, Cost of Illness, Envision, Pharmacology (medical), Genetics (clinical), Burden, Acute Porphyrias, Envision, Genetics & Heredity, Pain Research, PAIN, Hematology, Disease burden, General Medicine, LIVER-TRANSPLANTATION, Porphyria attack, Medicine, Research & Experimental, 6.1 Pharmaceuticals, Hemin, Chronic Pain, Life Sciences & Biomedicine, Quality of life, Clinical Trials and Supportive Activities, Pain, Burden, Porphyrias, Clinical Research, Acute hepatic porphyria, Genetics, MANAGEMENT, Humans, Peripheral Neuropathy, Givosiran, Other Medical and Health Sciences, Science & Technology, Porphyria, Chronic symptoms, Neurosciences, Evaluation of treatments and therapeutic interventions, Porphobilinogen Synthase, Acute Intermittent, Porphyrias, Hepatic, Porphyria, Acute Intermittent, Quality of Life, UPDATE, Digestive Diseases, ATTACKS

وصف الملف: Electronic; application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d3dcc4a34a20afd30da2fd2112dd67d6Test
https://hdl.handle.net/11380/1261337Test

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: Taro Yamashita, Jaclyn Franklin, Laura Obici, Julian D. Gillmore, Thomas H. Brannagan, Alejandra González-Duarte, Jihong Chen, Marianne T. Sweetser, Matthias Schilling, Peter D. Gorevic, Jing Jing Wang, Céline Labeyrie, David Adams, Hartmut Schmidt, Arnt V. Kristen, Teresa Coelho, John L. Berk, Senda Ajroud-Driss

المصدر: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Agência para a Sociedade do Conhecimento (UMIC)-FCT-Sociedade da Informação
instacron:RCAAP

مصطلحات موضوعية: Oncology, Adult, Male, medicine.medical_specialty, patisiran, Adolescent, hereditary transthyretin-mediated amyloidosis, 030204 cardiovascular system & hematology, 03 medical and health sciences, Polyneuropathies, Young Adult, 0302 clinical medicine, Quality of life, RNA interference, Internal medicine, APOLLO, Internal Medicine, Medicine, Humans, Prealbumin, Norfolk QOL-DN, In patient, RNA, Small Interfering, Aged, Aged, 80 and over, Amyloid Neuropathies, Familial, biology, business.industry, Amyloidosis, Middle Aged, medicine.disease, Placebo Effect, humanities, Multisystem disease, Transthyretin, quality of life, Mutation, biology.protein, Quality of Life, Female, business, 030217 neurology & neurosurgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::02d16f0eb1c0040f9c7946c39acb5c5cTest

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: Alejandra González-Duarte, Erhan Berber, Marianne T. Sweetser, Davide Adams, Teresa Coelho, Elizabeth Mauricio, Jing Jing Wang, Marco Luigetti, Michael Polydefkis, Matthew T. White, Thomas H. Brannagan, Jonas Wixner, Hartmut Schmidt

المصدر: Journal of the Neurological Sciences. 429:117766

مصطلحات موضوعية: medicine.medical_specialty, Neurology, business.industry, Amyloidosis, medicine, In patient, Neurology (clinical), Extension (predicate logic), Open label, medicine.disease, business, Surgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8685d85d0e9132d669c70b2604f03338Test
https://doi.org/10.1016/j.jns.2021.117766Test

المؤلفون: Teresa Coelho, John L. Berk, Isabel Conceição, Céline Tard, Madeline Merkel, Jing Jing Wang, Hartmut Schmidt, Dianna Quan, Alejandra González-Duarte, Márcia Waddington-Cruz, Cécile Cauquil, Michael Polydefkis, Mitsuharu Ueda, Michelle L. Mauermann, Arnt V. Kristen, Marianne T. Sweetser, Emre Aldinc, Jihong Chen, David Adams

المصدر: Journal of Neurology. 267:713-714

مصطلحات موضوعية: Oncology, medicine.medical_specialty, Neurology, biology, business.industry, Amyloidosis, medicine.disease, Transthyretin, RNA interference, Internal medicine, biology.protein, medicine, In patient, Neurology (clinical), business, Neuroradiology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::fd66aab996a7cb3e1ee81afac36eb4adTest
https://doi.org/10.1007/s00415-020-09715-5Test

المؤلفون: Marianne T. Sweetser, Ludwig Kappos, Krzysztof Selmaj, Bhupendra Khatri, Kimberly Umans, Gavin Giovannoni, Steven J. Greenberg, Jacob Elkins, Peter McCroskery, Ralf Gold

المصدر: Multiple Sclerosis and Related Disorders. 9:36-46

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Daclizumab, MedDRA, Antibodies, Monoclonal, Humanized, Disability Evaluation, 03 medical and health sciences, Multiple Sclerosis, Relapsing-Remitting, 0302 clinical medicine, Internal medicine, Clinical endpoint, medicine, Humans, Cumulative incidence, 030212 general & internal medicine, Adverse effect, business.industry, Multiple sclerosis, General Medicine, medicine.disease, Discontinuation, Surgery, Treatment Outcome, Neurology, Tolerability, Immunoglobulin G, Female, Neurology (clinical), business, Immunosuppressive Agents, 030217 neurology & neurosurgery, Follow-Up Studies, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8a97ba158857963aa7a83995b7e57230Test
https://doi.org/10.1016/j.msard.2016.05.010Test