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1دورية أكاديمية
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
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2
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
instnameمصطلحات 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 -
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المؤلفون: Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose, Laurent Servais, Hui Xiong, Edmar Zanoteli, Giovanni Baranello, Claudio Bruno, John W Day, Nicolas Deconinck, Andrea Klein, Eugenio Mercuri, Dmitry Vlodavets, Yi Wang, Angela Dodman, Muna El-Khairi, Ksenija Gorni, Birgit Jaber, Heidemarie Kletzl, Eleni Gaki, Paulo Fontoura, Basil T Darras, Joseph J Volpe, John Posner, Ulrich Kellner, Rosaline Quinlivan, Marianne Gerber, Omar Khwaja, Renata S Scalco, Timothy Seabrook, Armin Koch, Irina Balikova, Inge Joniau, Geraldine Accou, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Rodrigo de Holanda Mendonça, Ciro Matsui Jr, Ana Letícia Fornazieri Darcie, Cleide Machado, Maria Kiyoko Oyamada, Joyce Martini, Graziela Polido, Juliana Rodrigues Iannicelli, Juliana Caires de Oliveira Achili Ferreira, Chaoping Hu, Xiaomei Zhu, Chen Qian, Li Shen, Hui Li, Yiyun Shi, Shuizhen Zhou, Ying Xiao, Zhenxuan Zhou, Sujuan Wang, Tian Sang, Cuijie Wei, Hui Dong, Yiwen Cao, Jing Wen, Wenzhu Li, Lun Qin, Nina Barisic, Ivan Celovec, Martina Galiot Delic, Petra Kristina Ivkic, Nenad Vukojevic, Ivana Kern, Boris Najdanovic, Marin Skugor, Josipa Tomas, Odile Boespflug-Tanguy, Silvana De Lucia, Andrea Seferian, Emmanuel Barreau, Nabila Mnafek, Helene Peche, Allison Grange, Diem Trang Nguyen, Darko Milascevic, Shotaro Tachibana, Emanuela Pagliano, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, Marta Vigano, Claudia Dosi, Riccardo Zanin, Veronica Schembri, Noemi Brolatti, Giuseppe Rao, Elisa Tassara, Simone Morando, Paola Tacchetti, Marina Pedemonte, Enrico Priolo, Lorenza Sposetti, Giacomo Pietro Comi, Alessandra Govoni, Silvia Gabriella Osnaghi, Valeria Minorini, Francesca Abbati, Federica Fassini, Michaela Foa, Amalia Lopopolo, Marika Pane, Concetta Palermo, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, Guglielmo D'Amico, Lorenzo Orazi, Giorgia Coratti, Daniela Leone, Antonaci Laura, Roberto De Sanctis, Beatrice Berti, Naoki Kimura, Yasuhiro Takeshima, Hideki Shimomura, Tomoko Lee, Fumi Gomi, Takanobu Morimatsu, Toru Furukawa, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Sandra Modrzejewska, Anna Lemska, Evgenia Melnik, Svetlana Artemyeva, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Elena Litvinova, Cornelia Enzmann, Elea Galiart, Konstantin Gugleta, Christine Wondrusch Haschke, Haluk Topaloglu, Ibrahim Oncel, Nesibe Eroglu Ertugrul, Bahadir Konuskan, Bora Eldem, Sibel Kadayifçilar, Ipek Alemdaroglu, Seher Sari, Neslihan Bilgin, Aynur Ayse Karaduman, Fatma Gokcem Yildiz Sarikaya, Robert J Graham, Partha Ghosh, David Casavant, Alexis Levine, Rachael Titus, Amanda Engelbrekt, Lucia Ambrosio, Anne Fulton, Anna Maria Baglieri, Courtney Dias, Elizabeth Maczek, Amy Pasternak, Shannon Beres, Tina Duong, Richard Gee, Sally Young
المساهمون: Masson, R., Mazurkiewicz-Beldzinska, M., Rose, K., Servais, L., Xiong, H., Zanoteli, E., Baranello, G., Bruno, C., Day, J. W., Deconinck, N., Klein, A., Mercuri, E., Vlodavets, D., Wang, Y., Dodman, A., El-Khairi, M., Gorni, K., Jaber, B., Kletzl, H., Gaki, E., Fontoura, P., Darras, B. T., Volpe, J. J., Posner, J., Kellner, U., Quinlivan, R., Gerber, M., Khwaja, O., Scalco, R. S., Seabrook, T., Koch, A., Balikova, I., Joniau, I., Accou, G., Tahon, V., Wittevrongel, S., De Vos, E., de Holanda Mendonca, R., Matsui Jr, C., Fornazieri Darcie, A. L., Machado, C., Kiyoko Oyamada, M., Martini, J., Polido, G., Rodrigues Iannicelli, J., Caires de Oliveira Achili Ferreira, J., Hu, C., Zhu, X., Qian, C., Shen, L., Li, H., Shi, Y., Zhou, S., Xiao, Y., Zhou, Z., Wang, S., Sang, T., Wei, C., Dong, H., Cao, Y., Wen, J., Li, W., Qin, L., Barisic, N., Celovec, I., Galiot Delic, M., Ivkic, P. K., Vukojevic, N., Kern, I., Najdanovic, B., Skugor, M., Tomas, J., Boespflug-Tanguy, O., De Lucia, S., Seferian, A., Barreau, E., Mnafek, N., Peche, H., Grange, A., Trang Nguyen, D., Milascevic, D., Tachibana, S., Pagliano, E., Bianchi Marzoli, S., Santarsiero, D., Garcia Sierra, M., Tremolada, G., Arnoldi, M. T., Vigano, M., Dosi, C., Zanin, R., Schembri, V., Brolatti, N., Rao, G., Tassara, E., Morando, S., Tacchetti, P., Pedemonte, M., Priolo, E., Sposetti, L., Comi, G. P., Govoni, A., Osnaghi, S. G., Minorini, V., Abbati, F., Fassini, F., Foa, M., Lopopolo, A., Pane, M., Palermo, C., Pera, M. C., Amorelli, G. M., Barresi, C., D'Amico, G., Orazi, L., Coratti, G., Leone, D., Laura, A., De Sanctis, R., Berti, B., Kimura, N., Takeshima, Y., Shimomura, H., Lee, T., Gomi, F., Morimatsu, T., Furukawa, T., Stodolska-Koberda, U., Waskowska, A., Kolendo, J., Sobierajska-Rek, A., Modrzejewska, S., Lemska, A., Melnik, E., Artemyeva, S., Leppenen, N., Yupatova, N., Monakhova, A., Papina, Y., Shidlovsckaia, O., Litvinova, E., Enzmann, C., Galiart, E., Gugleta, K., Wondrusch Haschke, C., Topaloglu, H., Oncel, I., Ertugrul, N. E., Konuskan, B., Eldem, B., Kadayifcilar, S., Alemdaroglu, I., Sari, S., Bilgin, N., Karaduman, A. A., Sarikaya, F. G. Y., Graham, R. J., Ghosh, P., Casavant, D., Levine, A., Titus, R., Engelbrekt, A., Ambrosio, L., Fulton, A., Baglieri, A. M., Dias, C., Maczek, E., Pasternak, A., Beres, S., Duong, T., Gee, R., Young, S.
مصطلحات موضوعية: Muscular Atrophy, Spinal, Settore MED/26 - NEUROLOGIA, Pyrimidines, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Settore MED/48 - SCIENZE INFERMIERISTICHE E TECNICHE NEURO-PSICHIATRICHE E RIABILITATIVE, Humans, Infant, Neurology (clinical), Spinal Muscular Atrophies of Childhood, Azo Compounds, spinal muscular atrophy
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a209c925835005e2afe4ee89b6b5b41cTest
https://hdl.handle.net/10807/226647Test -
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المؤلفون: Maria Mazurkiewicz-Bełdzińska, Marianne Gerber, Heidemarie Kletzl, R. Masson, Andrea Klein, Edmar Zanoteli, Ksenija Gorni, Basil T. Darras, Kristy Rose, Giovanni Baranello, Laurent Servais, H. Xiong, D. Vlodavets, A. Dodman, M. El-Khairi, Renata S. Scalco
المصدر: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics.
مصطلحات موضوعية: business.industry, Anesthesia, medicine, Spinal muscular atrophy, medicine.disease, SMA, business
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::89e5acac415f97f435c35a50acd74960Test
https://doi.org/10.1055/s-0041-1739636Test -
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المؤلفون: Leon Aarons, Heidemarie Kletzl, Yumi Cleary, Michael Gertz, Andreas Günther, Katja Heinig, Aleksandra Galetin, Kayode Ogungbenro, Paul Grimsey
المصدر: Fukushima (Cleary), Y, Gertz, M, Grimsey, P, Günther, A, Heinig, K, Ogungbenro, K, Aarons, L, Galetin, A & Kletzl, H 2021, ' Model-based drug-drug interaction extrapolation strategy from adults to children – risdiplam in pediatric patients with spinal muscular atrophy ', Clinical Pharmacology & Therapeutics . https://doi.org/10.1002/cpt.2384Test
مصطلحات موضوعية: Adult, Male, Oncology, Physiologically based pharmacokinetic modelling, medicine.medical_specialty, Adolescent, CYP3A, Midazolam, Models, Biological, Muscular Atrophy, Spinal, Young Adult, Pharmacokinetics, In vivo, Internal medicine, Humans, Medicine, Drug Interactions, Pharmacology (medical), Child, Pharmacology, business.industry, Infant, Newborn, Area under the curve, Infant, Spinal muscular atrophy, SMA, medicine.disease, Pyrimidines, Neuromuscular Agents, Child, Preschool, Cytochrome P-450 CYP3A Inhibitors, Female, business, Azo Compounds, medicine.drug
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::60d9913e9af5e9910daa01792db02396Test
https://www.research.manchester.ac.uk/portal/en/publications/modelbased-drugdrug-interaction-extrapolation-strategy-from-adults-to-children--risdiplam-in-pediatric-patients-with-spinal-muscular-atrophyTest(c85742c4-c14d-419d-9311-c599b740e7f5).html -
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المؤلفون: Heidemarie Kletzl, D. Vlodavets, Marianne Gerber, Sabine Fuerst-Recktenwald, Maria Mazurkiewicz-Bełdzińska, Basil T. Darras, M. El-Khairi, Giovanni Baranello, Ksenija Gorni, Renata S. Scalco, Riccardo Masson, H. Xiong, Kristy Rose, Edmar Zanoteli, Laurent Servais
المصدر: Web of Science
مصطلحات موضوعية: Pediatrics, medicine.medical_specialty, business.industry, Gross motor skill, Spinal muscular atrophy, medicine.disease, Sitting, SMA, Bayley Scales of Infant Development, 03 medical and health sciences, 0302 clinical medicine, 030228 respiratory system, Tolerability, medicine, Clinical endpoint, 030212 general & internal medicine, Toddler, business
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ac31266581f9dd75f3911be295696a45Test
https://doi.org/10.1183/13993003.congress-2020.1172Test -
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المؤلفون: M. Al-Muhaizea, A. Prufer, Marianne Gerber, Y. Wang, Leslie Nelson, Edmar Zanoteli, Richard S. Finkel, Michelle A. Farrar, L. Servais, Renata S Scalco, Heidemarie Kletzl, E. Bertini, M. El-Khairi, Ksenija Gorni, L. Palfreeman
المصدر: Neuromuscular Disorders. 31:S48
مصطلحات موضوعية: Pathology, medicine.medical_specialty, biology, business.industry, Spinal muscular atrophy, Rainbowfish, medicine.disease, biology.organism_classification, SMA, Neurology, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::e526d2e30c62cb67f40bf5f07beb4359Test
https://doi.org/10.1016/j.nmd.2021.07.021Test -
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المؤلفون: Heidemarie Kletzl, Odile Boespflug-Tanguy, Nicolas Deconinck, Riccardo Masson, D. Vlodavets, Giovanni Baranello, J. Day, Kristy Rose, H. Xiong, Edmar Zanoteli, Ksenija Gorni, Eugenio Mercuri, E. Gaki, L. Servais, A. Dodman, M. El-Khairi, Marianne Gerber, Basil T. Darras, Andrea Klein, Maria Mazurkiewicz-Bełdzińska
المصدر: Neuromuscular Disorders. 31:S134
مصطلحات موضوعية: Pathology, medicine.medical_specialty, Neurology, business.industry, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), Spinal muscular atrophy, medicine.disease, SMA, business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::3a82a7c5134952d977f87858d3968a50Test
https://doi.org/10.1016/j.nmd.2021.07.303Test -
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المؤلفون: D. Vlodavets, Kristy Rose, Edmar Zanoteli, L. Servais, Renata S Scalco, Andrea Klein, Heidemarie Kletzl, Marianne Gerber, Basil T. Darras, M. El-Khairi, Maria Mazurkiewicz-Bełdzińska, Riccardo Masson, J. Day, Giovanni Baranello, Ksenija Gorni, Eugenio Mercuri, O. Bloespflug-Tanguy, H. Xiong, Nicolas Deconinck, A. Dodman
المصدر: Neuromuscular Disorders. 30:S126
مصطلحات موضوعية: Neurology, business.industry, Anesthesia, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), Spinal muscular atrophy, medicine.disease, SMA, business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::83865779a0847b36f213b06279b35686Test
https://doi.org/10.1016/j.nmd.2020.08.270Test -
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المؤلفون: Agnès Poirier, Lutz Mueller, Heidemarie Kletzl, Chien-Ping Ko, Stefan Kustermann, Omar Khwaja, Hasane Ratni, Zhihua Feng, Johan Bylund, Bjoern Jacobsen, Michael Reutlinger, Melanie Guérard, Emmanuel Pinard, Sergey Paushkin, John D. Baird, Nikolai Naryshkin, Stefanie Bendels, Anne Marquet, Nora Denk, Xin Zhao, Philippe Jablonski, Martin Ebeling, Luke Green, Friedrich Metzger, Andreas Zeller, Barbara Mueller, Karen S. Chen, Marla Weetall
المصدر: Journal of medicinal chemistry. 61(15)
مصطلحات موضوعية: 0301 basic medicine, Drug discovery, Chemistry, Gene splicing, RNA Splicing, Survival of motor neuron, Spinal muscular atrophy, medicine.disease, Bioinformatics, SMA, Clinical trial, Muscular Atrophy, Spinal, Survival of Motor Neuron 2 Protein, 03 medical and health sciences, 030104 developmental biology, Pyrimidines, RNA splicing, Drug Discovery, medicine, Molecular Medicine, Animals, Humans, Dosing, Safety, Azo Compounds
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7d9e69d504357bfed8927794453d1858Test
https://pubmed.ncbi.nlm.nih.gov/30044619Test