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1
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 -
2
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 -
3
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 -
4
المؤلفون: Heidemarie Kletzl, A. Höflich, Friedrich Metzger, Eginhard Schick, Christoph Wandel, Norbert Bleich, C. Höflich, R.F. Staack, Andreas Guenther, Jan Frystyk
المصدر: Kletzl, H, Guenther, A, Höflich, A, Höflich, C, Frystyk, J, Staack, R F, Schick, E, Wandel, C, Bleich, N & Metzger, F 2017, ' First-in-man study with a novel PEGylated recombinant human insulin-like growth factor-I ', Growth Hormone & I G F Research, vol. 33, pp. 9-16 . https://doi.org/10.1016/j.ghir.2017.01.001Test
مصطلحات موضوعية: Male, 0301 basic medicine, medicine.medical_specialty, Maximum Tolerated Dose, Erythema, Endocrinology, Diabetes and Metabolism, Hypoglycemia, Polyethylene Glycols, 03 medical and health sciences, Subcutaneous injection, 0302 clinical medicine, Endocrinology, Double-Blind Method, Pharmacokinetics, Internal medicine, medicine, Humans, Tissue Distribution, Insulin-Like Growth Factor I, Growth Substances, First-in-man study, Dose-Response Relationship, Drug, business.industry, Prognosis, medicine.disease, Recombinant Proteins, 030104 developmental biology, Tolerability, Pharmacodynamics, PEGylation, Female, medicine.symptom, business, 030217 neurology & neurosurgery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::830323b68af1ea8d8ea70fdc93cf9300Test
https://doi.org/10.1016/j.ghir.2017.01.001Test -
5
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 -
6
المؤلفون: Riccardo Masson, K. Gelblin, J. Day, Heidemarie Kletzl, Andrea Klein, Ksenija Gorni, Y. Cleary, Nicolas Deconinck, L. Servais, Christian Czech, C. Nguyen, Giovanni Baranello, T. Seabrook, M. El-Khairi, Marianne Gerber, Eugenio Mercuri, Basil T. Darras
المصدر: Neuromuscular Disorders. 29:S184
مصطلحات موضوعية: Neurology, business.industry, Anesthesia, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), Spinal muscular atrophy, medicine.disease, business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::c86208b936e4a2174fabfc6eb5c0050eTest
https://doi.org/10.1016/j.nmd.2019.06.515Test -
7
المؤلفون: J. Day, D. Kraus, Marianne Gerber, Ksenija Gorni, Y. Cleary, Eugenio Mercuri, Dirk Fischer, Heidemarie Kletzl, W. Yeung, Claudia A. Chiriboga
المصدر: Neuromuscular Disorders. 29:S187
مصطلحات موضوعية: medicine.medical_specialty, business.industry, Urology, Spinal muscular atrophy, SMA, medicine.disease, Neurology, Pharmacodynamics, Pediatrics, Perinatology and Child Health, Medicine, In patient, Nusinersen, Neurology (clinical), business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::70dc6908b7231adac2a35353e040b0c1Test
https://doi.org/10.1016/j.nmd.2019.06.525Test -
8
المؤلفون: S. Sturm, John W. Day, Ksenija Gorni, Y. Cleary, Dirk Fischer, L. Servais, Eugenio Mercuri, Heidemarie Kletzl, Janbernd Kirschner, Maria Carmela Pera, Nicolas Deconinck, Andrea Klein, Riccardo Masson, Nathalie Goemans, Christian Czech, Andreas Günther, Claudia A. Chiriboga, Omar Khwaja, Basil T. Darras, Giovanni Baranello
المصدر: Neuromuscular Disorders. 28:S109-S110
مصطلحات موضوعية: 0301 basic medicine, Oncology, medicine.medical_specialty, business.industry, SMA, 03 medical and health sciences, 030104 developmental biology, 0302 clinical medicine, Neurology, Internal medicine, Pediatrics, Perinatology and Child Health, Medicine, Neurology (clinical), business, 030217 neurology & neurosurgery, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::34ea05adadbc5deddbf33c263194249cTest
https://doi.org/10.1016/j.nmd.2018.06.307Test -
9
المؤلفون: M. Alexander, D. Kraus, Omar Khwaja, A. Cho, Dirk Fischer, H. Abdallah, Christian Czech, Anne Marquet, Heidemarie Kletzl, G. Armstrong, L. Mueller, Claudia A. Chiriboga, Ksenija Gorni, T. Seabrook, Eugenio Mercuri
المصدر: Neuromuscular Disorders. 27:S209
مصطلحات موضوعية: medicine.medical_specialty, business.industry, Targeting therapy, Safety tolerability, Spinal muscular atrophy, medicine.disease, Surgery, Neurology, Pharmacokinetics, Open label study, Internal medicine, Pharmacodynamics, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), business, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::6e1db3fdca78b639cd568aa5fc94d4fbTest
https://doi.org/10.1016/j.nmd.2017.06.416Test -
10
المؤلفون: Omar Khwaja, Eugenio Mercuri, H. Abdallah, Giovanni Baranello, Maria Carmela Pera, Janbernd Kirschner, Christian Czech, Nathalie Goemans, L. Servais, G. Armstrong, L. Mueller, Ksenija Gorni, Heidemarie Kletzl, S. Sturm, T. Seabrook, Anne Marquet, D. Kraus
المصدر: Neuromuscular Disorders. 27:S209
مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, business.industry, Spinal muscular atrophy, medicine.disease, Surgery, 03 medical and health sciences, 030104 developmental biology, 0302 clinical medicine, Neurology, Pediatrics, Perinatology and Child Health, Medicine, In patient, Neurology (clinical), business, 030217 neurology & neurosurgery, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::352d44eb117b17bb0bf47ee7aa2c57fdTest
https://doi.org/10.1016/j.nmd.2017.06.415Test