-
1
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 -
2
المؤلفون: David Adams, Michael Polydefkis, Alejandra González-Duarte, Jonas Wixner, Arnt V Kristen, Hartmut H Schmidt, John L Berk, Inés Asunción Losada López, Angela Dispenzieri, Dianna Quan, Isabel M Conceição, Michel S Slama, Julian D Gillmore, Theodoros Kyriakides, Senda Ajroud-Driss, Márcia Waddington-Cruz, Michelle M Mezei, Violaine Planté-Bordeneuve, Shahram Attarian, Elizabeth Mauricio, Thomas H Brannagan, Mitsuharu Ueda, Emre Aldinc, Jing Jing Wang, Matthew T White, John Vest, Erhan Berber, Marianne T Sweetser, Teresa Coelho, Giuseppe Vita, Vincenzo Rizzo, Massimo Russo, Anna Mazzeo, Luca Gentile, Caitlin Brueckner, Victoria Lazzari, Janice Wiesman, Douglas DeLong, Jennifer Victory, James Dalton, John May, Catherine Gilmore, Saran Diallo, Emilien Delmont, Jean Pouget, Annie Verschueren, Aude-Marie Grapperon, Emmanuelle Campana-Salort, Ana Lopes, Filipa Lamas, Carlos Neves, Jose Castro, Pedro Pereira, Isabel Castro, Ana Franco, Miguel Oliveira Santos, Conceição de Azevedo Coutinho, Catarina Falcao de Campos, Antonio Hipólito Reis, Nuno Correia, Javier M Perez, Ana Martins da Silva, Cristina Alves, Marcio Cardoso, Katia Valdrez, Julia R Monte, Bernardete Pessoa, Nadia Guimaraes, Monica Freitas, Joana Ramalho, Natalia Ferreira, Daisuke Kuzume, Celine Tard, Nawal Waucquier, Isabelle Rougeaux, Sylvie Brice, Emmanuelle Kasprzyk, Elise Elrezzi, Sayah Meguig, Eric Hachulla, Clement Gauvain, Maria-Claire Migaud-Chervy, Dominique Deplanque, Elsa Jozefowicz, Loic Lebellec, Line Balaya-Gouraya, Nathalie Jehan Lacour, Halima Bournane, Nathalie Martin, Mongia Elabed, Niamey Sacko, Yasmine Boubrit, Amina Gaouar, Fetra Rakotondratafika, Marie Théaudin-Saliou, Cécile Cauquil-Michon, Celine Labeyrie, Adeline Not, Abdallah Al-Salameh, Anne-Lise Lecoq, Maeva Stephant, Andoni Echaniz-Laguna, Laurent Becquemont, Guillemette Beaudonnet, Vincent Algalarrondo, Ludivine Eliahou, Antoine Rousseau, Aissatou Signate, Emeline Berthelot, Jocelyn Inamo, Laetitia Vervoitte, Cecile Focseneanu, Thierry Gendre, Raphaele Arrouasse, Samar S. Ayache, Laura Ernande, Philippe Le Corvoisier, Hayet Salhi, Ariane Choumert, Vincent Ehinger, Julie Ruiz, Cyril Charlin, Thomas Megelin, Thomas H Brannagan III, Raisy Fayerman, Arreum Kim, Allan Paras, Leidy J Gonzalez, Steven Tsang, Fernanda Wajnsztajn, Jeffrey Shije, Christina Ulane, Inna Kleyman, Louis Weimer, Comana Cioroiu, Sakis Lambrianides, Rana Abu-Manneh, Eleni Zamba-Papanicolaou, Petros Agathangelou, Eleni Leonidou, Satoshi Tada, Akemi Fujita, Masahiro Nagai, Rina Ando, Yuko Hosokawa, Yuki Yamanishi, J. Scott Overcash, Elena Giardino, Leslie Boyer, Lien Dang, An Le, Tyler Nguyen, Lien Giang, Peter Sellers, Leyla Tran, Nghi Truong, Maita Vinas, Nicole Hrkman, Sarah Miller, David Nguyen, Ashley Smith, Helen Pu, Steve Li, Thao Vuong, Holly Dioso, Sinikka Green, Kia Lee, Hanh Chu, Michael Waters, Derya J Coskun, Karla A Zepeda, William O'Riordan, Laura Obici, Andrea Cortese, Alessandro Lozza, Giampaolo Merlini, Vittorio Rosti, Mario Sabatelli, Giulia Bisogni, Daniela Bernardo, Marco Luigetti, Andrea Di Paolantonio, Valeria Guglielmino, Angela Romano, Hans Nienhuis, Janita Bulthuis-Kuiper, Olga Gerk, Hannah Ulbricht, Lenka Taylor, Eva Meyle, Natalia Kleinschmidt, David Meyrath, Simone Noe-Schwenn, Ulrike Meng, Ralf Bauer, Fabian aus dem Siepen, Selina Hein, Tetsuya Takahashi, Tomohiko Oshita, Yoko Koujin, Shuichiro Neshige, Tomohisa Nezu, Akiko Segawa, Hiroki Ueno, Hiroyuki Morino, Josep M Campistol, Lida Maria Rodas Marin, Josep Miquel Blasco Pelicano, Lucía Galán Dávila, Marta Palacios, Vanesa Pytel Cordoba, Antonio Guerrero Sola, Alejandro Horga, Julián García Feijoo, Leopoldo Perez de Isla, Wilson Marques Júnior, Mariana Moscardini, Debora Cristina Litcanov, Ana Flavia Viera Lima, Leonardo Rodrigues, Barbara Marques Coutinho, Carolina Lavigne Moreira, Vanessa Daccach Marques, Francisco Munoz Beamud, Álvaro Gragera Martínez, Cristina Borrachero, Eugenia Cisneros Barroso, Adrián Rodríguez Rodríguez, Monica Sanz, Elena Rigo Oliver, Juan González Moreno, Jose M Gamez Martinez, Cristina Descals, Mercedes Uson, Francisco Jose Vega, Antoni Figuerola, Carles Montala, Moises Dias da Silva, Renata Gervais de Santa Rosa, Luiz Felipe Pinto, Marcus Vinicius Pinto, Amanda Cardoso Berensztejn, Fabio Barroso, Andrea Lautre, Lucas G Orellana, Maria Alejandra González-Duarte Briseño, Karla Cárdenas-Soto, Brenda Poled Jiménez López, Sandra Lorena Pérez-Castañeda, Carlos Gerardo Cantú Brito, David Rivera de la Parra, Jose Pablo Hernandez Reyes, Maria del Mar Saniger Alba, Elia Criollo Mora, Yesim Parman, Kus Jülide Rezzan, Erdi Sahin, Nail G Serbest, Hacer Durmus, Arman Cakar, Nuriye Ilknur Tugal Tutkun, Sacit Karamursel, Ali Elitok, Nermin G Sirin Inan, Emre Altinkurt, Jing Ye, Adriane C Allen, Vinay Chaudhry, Raquel Jarrett, Neil Bressler, Kathleen L Burks, Qingfeng Liu, Mohammad Khoshnoodi, Daniel P Judge, Geno Vista, Syed Mahmood Shah, Hirotoshi Hamaguchi, Junko Oda, Emi Fukase, Ikuko Taniguchi, Tetsuya Oda, Hironobu Endo, Masahiro Shimomura, Kimitaka Katanazaka, Shusuke Koto, Takahiro Nakano, Christof Scheid, Andreas Zueiter, Lars Pester, Doreen Walter, Betül Özdemir, Lukas F Frenzel, Udo Holtick, Jeeyoung Oh, Hee Jin Kim, Hyun Jin Shin, Kyomin Choi, Taro Yamashita, Teruaki Masuda, Yohei Misumi, Akihiko Ueda, Keiichi Nakahara, Akiko Yorita, Seiko Tsuruhisa, Takayuki Taniwaki, Masaya Harada, Taiga Moritaka, Naonori Sakurada, Elizabeth A Mauricio, Amber Baskin, Elliot Dimberg, Amie Fonder, Miriam Hobbs, Stephen J Russell, Peter Dyck, Wilson Gonsalves, Nelson Leung, Thomas E Witzig, Steven R Zeldenrust, Lisa Hwa, Prashant Kapoor, Shaji K Kumar, Yi Lin, John A Lust, Vincent S Rajkumar, David Dingli, Morie A Gertz, Ronald Go, Suzanne R Hayman, Samir Dalia, Esmeralda Carrillo, Peter Gorevic, Garnette Mason, Chi-Chao Chao, Ming-Jen Lee, Jen-Jen Su, Sung-Tsang Hsieh, Li-Kai Tsai, Shin-Joe Yeh, Chih-Chao Yang, Senda Ajroud-Driss Ajroud-Driss, Patricia Casey, Benjamin C Joslin, Miriam Freimer, Alison Sankey, Amanda Kenepp, Sarah Heintzman, Samantha LoRusso, Youichi Hokezu, Byoung-Joon Kim, JuHyeon Kim, Ga Yeon Lee, Eun Bin Cho, Eun-Seok Jeon, Ju-Hong Min, Jin Myoung Seok, Hye Lim Lee, Jae Hong Park, Yoshiki Sekijima, Chinatsu Miyazawa, Nagaaki Kato, Dai Kishida, Akiyo Hineno, Minori Kodaira, Tsuneaki Yoshinaga, Teruyoshi Miyahara, Akira Imai, Kazuhiko Matsumoto, Kon-Ping Lin, Yi-Chung Lee, Malin Falk, Bjorn Pilebro, Ole Suhr, Per Lindqvist, Karin Soderberg, Fatima Pedrosa-Domellöf, Intissar Anan, Erik Nordh, Ivaylo Tournev, Sashka Zhelyazkova-Glaveeva, Zheyna Cherneva, Staiko Sarafov, Teodora Chamova, Sylvia Cherninkova-Gopina, Frauke Friebel, Andree Zibert, Natasa Mihailovic, Friederike Schubert, Elena Vorona, Larissa Lahme, Anna Huesing-Kabar, Matthias Schilling, Iyad Kabar, Ana Martinez-Naharro, Liza Chacko, Oliver Cohen, Steven Law, Tamer Rezk, Helen J Lachmann, Brianna Blume, Stacy Dixon, Soon Chai Low, Soo Looi Chan, He Eng Li Lim, Khean Jin Goh, Deborah Kraus, Kristin Jack, N. Kevin Wade, Glenn Lopate, Brittany Zwijack, Julaine Florence, R. Brian Sommerville, Graeme Stewart, Julie Ryder, Linda Mekhael, Mark Taylor, Daniel Suan, Karen Wells, Paula Stone, Amenze Itoya, Mercy Owusu-Sekyere, Desmond Thai, Ilonah Chahine, Salve Pedrosa, Thi Hoa (Therese) Do
المساهمون: Repositório da Universidade de Lisboa
المصدر: The Lancet Neurology. 20:49-59
مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Drug-Related Side Effects and Adverse Reactions, 030204 cardiovascular system & hematology, Placebo, Severity of Illness Index, Polyneuropathies, 03 medical and health sciences, 0302 clinical medicine, Quality of life, Internal medicine, Outcome Assessment, Health Care, Severity of illness, medicine, Humans, Prealbumin, RNA, Small Interfering, Infusions, Intravenous, Adverse effect, Aged, Amyloid Neuropathies, Familial, business.industry, Middle Aged, medicine.disease, Interim analysis, amyloidosis, transthyretin, polyneuropathy, patisiran, OLE study, Clinical trial, Female, Neurology (clinical), business, Polyneuropathy, 030217 neurology & neurosurgery, Progressive disease
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ce6a19ce91101df4f11e95eb6d009161Test
https://doi.org/10.1016/s1474-4422Test(20)30368-9 -
3
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 -
4
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 -
5
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 -
6
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 -
7
المؤلفون: Marianne T. Sweetser, Gabriel J. Robbie, Xiaoping Zhang, Varun Goel, Valerie A. Clausen, Husain Attarwala
المصدر: Journal of Clinical Pharmacology
مصطلحات موضوعية: Male, patisiran, Therapeutics, Pharmacology, Placebo, 030226 pharmacology & pharmacy, small interfering ribonucleic acid (siRNA), 03 medical and health sciences, Polyneuropathies, 0302 clinical medicine, Pharmacokinetics, Double-Blind Method, pharmacokinetics (PK), Medicine, Humans, Prealbumin, Pharmacology (medical), Dosing, RNA, Small Interfering, Adverse effect, Aged, Amyloid Neuropathies, Familial, biology, Dose-Response Relationship, Drug, business.industry, Amyloidosis, Middle Aged, pharmacodynamics (PD), medicine.disease, Transthyretin, RNAi Therapeutics, Treatment Outcome, Liver, 030220 oncology & carcinogenesis, Pharmacodynamics, Liposomes, biology.protein, Nanoparticles, Administration, Intravenous, Female, exposure‐response, business, Polyneuropathy, hereditary transthyretin‐mediated amyloidosis (hATTR)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9565a43aa8b1fa7cdcb2fe7d26f12920Test
https://pubmed.ncbi.nlm.nih.gov/31322739Test -
8
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 -
9
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 -
10
المؤلفون: Gerald G. Krueger, Mohamed Darif, Kim A. Papp, Bernard S. Goffe, Marianne T. Sweetser, Barry Ticho, Carmen Bozic, Sophia Lee, David Gratton
المصدر: Clinical Therapeutics. 27:1912-1921
مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Adolescent, Recombinant Fusion Proteins, Population, Alefacept, Infections, Antibodies, Neoplasms, Psoriasis, Internal medicine, medicine, Humans, Pharmacology (medical), education, Adverse effect, Aged, Aged, 80 and over, Pharmacology, Clinical Trials as Topic, education.field_of_study, business.industry, Incidence (epidemiology), Middle Aged, medicine.disease, Surgery, Clinical trial, Upper respiratory tract infection, Tolerability, Female, Dermatologic Agents, business, medicine.drug
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e1bbb30b00e2253ff2b5844dfd3de039Test
https://doi.org/10.1016/j.clinthera.2005.12.007Test