المؤلفون: Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group

المصدر: Neurology and Therapy, Vol 12, Iss 2, Pp 543-557 (2023)

مصطلحات موضوعية: Evrysdi, Pharmacodynamics, Risdiplam, Safety, Spinal muscular atrophy, Neurology. Diseases of the nervous system, RC346-429

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2193-8253Test; https://doaj.org/toc/2193-6536Test

الوصول الحر: https://doaj.org/article/cf9aa173befd4087b4e4bc7cce3e9e84Test

المؤلفون: Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group

المصدر: Neurology and Therapy, Vol 12, Iss 5, Pp 1799-1801 (2023)

مصطلحات موضوعية: Neurology. Diseases of the nervous system, RC346-429

وصف الملف: electronic resource

العلاقة: https://doaj.org/toc/2193-8253Test; https://doaj.org/toc/2193-6536Test

الوصول الحر: https://doaj.org/article/258bc786e54b4365b79dbe9dd14b5d86Test

المؤلفون: Mariacristina, Scoto, Claudio, Bruno, Dirk, Fischer, Janbernd, Kirschner, Eugenio, Mercuri, Imogen, Carruthers, Marianne, Gerber, Heidemarie, Kletzl, Teresa, Gidaro, Francesco, Muntoni

المصدر: Association of British Neurologists: Annual Meeting Abstracts 2023

الإتاحة: https://doi.org/10.1136/jnnp-2023-abn.177Test

المؤلفون: Jeffrey Statland, Katy Eichinger, Jasper Morrow, Giorgio Tasca, John Vissing, Angela Dodman, Birgit Jaber, Heidemarie Kletzl, Tammy McIver, Renata Scalco, Wai Yin Yeung, Eleni Gaki, Kathryn Wagner

المصدر: Sunday, April 23.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::644afd9c01d7db23276a291a99e9d27fTest
https://doi.org/10.1212/wnl.0000000000203667Test

المؤلفون: Basil Darras, Giovanni Baranello, Odile Boespflug-Tanguy, John Day, Nicholas Deconinck, Andrea Klein, Ricardo Masson, Maria Mazurkiewicz-Bełdzińska, Eugenio Mercuri, Kristy Rose, Dmitry Vlodavets, Hui Xiong, Edmar Zanoteli, Muna El-Khairi, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Laura Palfreeman, Angela Dodman, Laurent Servais

المصدر: Tuesday, April 25.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::6667a1d6337d215483d25780abd97f6aTest
https://doi.org/10.1212/wnl.0000000000203624Test

المؤلفون: Claudia Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Janbernd Kirschner, Mariacristina Scoto, Eugenio Mercuri, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Teresa Gidaro, Francesco Muntoni

المصدر: Tuesday, April 25.

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::3391cb63ab291b8f80329faa7c7a8a6aTest
https://doi.org/10.1212/wnl.0000000000203609Test

المؤلفون: Stephen L. Hauser, Amit Bar-Or, Martin S. Weber, Heidemarie Kletzl, Andreas Günther, Marianna Manfrini, Fabian Model, Francois Mercier, Claire Petry, Qing Wang, Harold Koendgen, Terence Smith, Ludwig Kappos

المصدر: Neurology(R) neuroimmunology & neuroinflammation, vol 10, iss 2

مصطلحات موضوعية: Multiple Sclerosis, Clinical Trials and Supportive Activities, Neurosciences, Neurodegenerative, Autoimmune Disease, Antibodies, Brain Disorders, Neurology, Recurrence, Clinical Research, Monoclonal, Neurological, Humans, Immunologic Factors, Neurology (clinical), Humanized, Interferon beta-1a

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2f42355c2e32e71d1f6394e52fde3da9Test
https://escholarship.org/uc/item/25z0k5ksTest

المؤلفون: Sean Yiu, Qing Wang, Francois Mercier, Marianna Manfrini, Harold Koendgen, Heidemarie Kletzl, Fabian Model

مصطلحات موضوعية: Nursing not elsewhere classified, Health and Community Services, Sociological Methodology and Research Methods, Confounding bias, exposure–response model, hazard ratio, weighting methods, outcome regression, selection bias

العلاقة: https://figshare.com/articles/journal_contribution/sj-pdf-2-smm-10_1177_09622802231212274_-_Supplemental_material_for_Estimating_drug_concentration_response_relationships_by_applying_causal_inference_methods_for_continuous_point_exposures_and_time-to-event_outcomes/24573798Test

الإتاحة: https://doi.org/10.25384/sage.24573798.v1Test
https://figshare.com/articles/journal_contribution/sj-pdf-2-smm-10_1177_09622802231212274_-_Supplemental_material_for_Estimating_drug_concentration_response_relationships_by_applying_causal_inference_methods_for_continuous_point_exposures_and_time-to-event_outcomes/24573798Test

المؤلفون: Eugenio Mercuri, Nicolas Deconinck, Elena S Mazzone, Andres Nascimento, Maryam Oskoui, Kayoko Saito, Carole Vuillerot, Giovanni Baranello, Odile Boespflug-Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera-Pruszczyk, Laurent Servais, Marianne Gerber, Ksenija Gorni, Omar Khwaja, Heidemarie Kletzl, Renata S Scalco, Hannah Staunton, Wai Yin Yeung, Carmen Martin, Paulo Fontoura, John W Day, Joseph J. Volpe, John Posner, Ulrich Kellner, Rosaline Quinlivan, Aurore Daron, Stéphanie Delstanche, Romain Bruninx, Fabian Dal Farra, Olivier Schneider, Irina Balikova, Patricia Delbeke, Inge Joniau, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Ingele Casteels, Liesbeth De Waele, Catherine Cassiman, Lies Prové, David Kinoo, Lisa Vancampenhout, Marleen Van Den Hauwe, Annelies Van Impe, Alexandra Prufer de Queiroz Campos Araujo, Aline Chacon Pereira, Flávia Nardes, Lorena Haefeli, Julia Rossetto, Marcos Ferreira Rebel, Jaqueline Almeida Pereira, Craig Campbell, Sapna Sharan, Wendy McDonald, Cheryl Scholtes, Jean Mah, Maria Sframeli, Angela Chiu, Jane Hagel, Raquel Beneish, Gaela Cariou-Palmer, Connie Pham, Daniela Toffoli, Stephanie Arpin, Sarah Turgeon Desilets, Yi Wang, Chaoping Hu, Jianfeng Huan, Chen Qian, Li Shen, Ying Xiao, Zhenxuan Zhou, Hui Li, Sujuan Wang, Hui Xiong, Xingzhi Chang, Hui Dong, Ying Liu, Tian Sang, Cuijie Wei, Jing Wen, Yiwen Cao, Xingyao Ly, Jingjing Zhao, Wenzhu Li, Lun Qin, Nina Barisic, Martina Galiot Delic, Petra Kristina Ivkic, Nenad Vukojevic, Ivana Kern, Boris Najdanovic, Marin Skugor, Teresa Gidaro, Andreea Seferian, Silvana De Lucia, Emmanuel Barreau, Nabila Mnafek, Marta Milkova Momtchilova, Helene Peche, Carole Valherie, Allison Grange, Charlotte Lilien, Darko Milascevic, Shotaro Tachibana, Claudia Ravelli, Ruxandra Cardas, Jessica Taytard, Guillaume Aubertin, Laure Vanden Brande, Jean-Baptiste Davion, Stephanie Coopman, Ikram Bouacha, Philippe Debruyne, Sabine Defoort, Gilles Derlyn, Florian Leroy, Loïc Danjoux, Julie Guilbaud, Isabelle Desguerre, Christine Barnérias, Michaela Semeraro, Dominique Bremond-Gignac, Lenaic Bruere, Maxence Rateaux, Élodie Deladrière, Virginie Germa, Yann Pereon, Sandra Mercie, Fanny Billaud, Lucie Le Goff, Guy Letellier, Aurélie Portefaix, Camille De-Montferrand, Laure Le-Goff, Stephanie Fontaine, Manel Saidi, Nabil Bouzid, Aurélie Barriere, Marie Tinat, Michelle Dreesbach, Wolf Lagréze, Bettina Michaelis, Fanni Molnar, Dorina Seger, Sibylle Vogt, Enrico Bertini, Adele D'Amico, Sergio Petroni, Anna Maria Bonetti, Adelina Carlesi, Irene Mizzoni, Claudio Bruno, Enrico Priolo, Giuseppe Rao, Simone Morando, Paola Tacchetti, Ambra Zuffi, Giacomo Pietro Comi, Roberta Brusa, Stefania Corti, Velardo Daniele, Alessandra Govoni, Francesca Magri, Valeria Minorini, Silvia Gabriella Osnaghi, Francesca Abbati, Federica Fassini, Michaela Foa, Amaqlia Lopopolo, Megi Meneri, Francesca Zoppas, Valeria Parente, Riccardo Masson, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, Marta Vigano, Riccardo Zanin, Laura Antonaci, Roberto de Sanctis, Marika Pane, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, Gugliemo D'Amico, Lorenzo Orazi, Giorgia Coratti, Kazuhiro Haginoya, Atsuko Kato, Yuko Morishita, Ryutaro Kira, Kiyomu Akiyama, Miwako Goto, Yujiro Mori, Misato Okamoto, Saki Tsutsui, Yuta Takatsuji, Aya Tanaka, Hirofumi Komaki, Miina Omori, Ippei Suzuki, Mizuki Takeuchi, Daisuke Todoroki, Seji Watanabe, Tomoko Matsubayashi, Emi Inakazu, Hiroe Nagura, Akira Suzuki, Manami Usui, Nobutsune Ishikawa, Yousuke Harada, Kenishi Fudeyasu, Kazuhiko Hirata, Kana Michiue, Kazuyuki Ueda, Junko Fujitani, Reiko Arakawa, Kozue Takano, Shigeko Yashiro, Maiko Seki, Nozomi Sano, Koji Fukuyama, Yuki Matsumoto, Hirofumi Miyazaki, Minoru Shibata, Kyoko Kobayashi, Yukie Nakamura, Yasuhiro Takeshima, Moe Kuma, Anna Fraczek, Maria Jedrzejowska, Anna Lusakowska, Agnieszka Czeszyk-Piotrowicz, Wojciech Hautz, Klaudia Rakusiewicz, Malgorzata Burlewicz, Zuzanna Gierlak-Wojcicka, Malwina Kepa, Adam Sikorski, Marcin Sobieraj, Maria Mazurkiewicz-Beldzinska, Anna Lemska, Sandra Modrzejewska, Mateusz Koberda, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Barbara Steinborn, Magdalena Dalz, Julia Grabowska, Wojciech Hajduk, Justyna Janasiewicz-Karachitos, Monika Klimas, Marcin Stopa, Ewa Gajewska, Beata Pusz, Dmitry Vlodavets, Evgenia Melnik, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Vedrana Milic Rasic, Vesna Brankovic, Ana Kosac, Olivera Djokic, Vesna Jakšic, Ana Pepic, Jelena Martinovic, Francina Munell Casadesus, Eduardo Tizzano, Nieves Martín Begué, Charlotte Wolley Dod, Olaia Subira, Bernat Planas Pascual, Esther Toro Tamargo, Marcos Madruga Garrido, José David Medina Romero, Marta Peña Salinas, Andrés Nascimento Osorio, Ana Díaz Cortés, Enrique Jiménez Gañan, Simone Dowon Suh, Julita Medina Cantillo, Obdulia Moya, Nuria Padros, Sandra Roca Urraca, Hugo Gonzalez Valdivia, Samuel Pascual Pascual, Sofía de Manuel, Susana Noval Martin, Paul Burnham, Sandra Espinosa, Mercedes Martinez Moreno, Haluk Topaloglu, Ibrahim Oncel, Nesibe Eroglu Ertugru, Bahadir Konuskan, Bora Eldem, Sibel Kadayifçilar, Ipek Alemdaroglu, Aynur Ayse Karaduman, Oznur Tunca Yilmaz, Neslihan Bilgin, Seher Sari, Claudia Chiriboga, John J. Lee, Donnielle Rome-Martin, John W. Day, Shannon Beres, Tina Duong, Richard Gee, Sally Dunaway Young, Sabine Fuerst-Recktenwald, Anne Marquet, Nicoletta Muelhardt, Dylan Trundell

المصدر: LANCET NEUROLOGY
r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname

مصطلحات موضوعية: Adult, Risdiplam, spinal muscular atrophy, Adolescent, Spinal Muscular Atrophies of Childhood, Settore MED/26 - NEUROLOGIA, Young Adult, Pyrimidines, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Double-Blind Method, Child, Preschool, Humans, Neurology (clinical), Child, Preschool, Azo Compounds, Aged

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::917024073c62a8e3c3c8eedaa6f463bbTest
https://doi.org/10.1016/s1474-4422Test(21)00367-7

المؤلفون: Agnès Poirier, Heidemarie Kletzl, Cordula Stillhart, Stephen Fowler, Kenichi Umehara, Andreas Guenther, Andreas Brink, Nicole A. Kratochwil, Dietrich Tuerck, Christophe Husser, Mark Savage, Mohammed Ullah, Lutz Mueller, Yumi Cleary, Katja Heinig

المصدر: Drug Metabolism and Disposition. 50:65-75

مصطلحات موضوعية: Pharmacology, Drug, Chemistry, In silico, media_common.quotation_subject, Pharmaceutical Science, Computational biology, Small molecule, Drug development, Pharmacokinetics, In vivo, Distribution (pharmacology), ADME, media_common

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::a42eb9cb10d47b49d0cb86fb0a1206acTest
https://doi.org/10.1124/dmd.121.000563Test