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1
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 -
2
المؤلفون: Jules A. A. C. Heuberger, Eugenio Mercuri, Katja Heinig, Russell Kinch, Andreas Günther, Irene Gerlach, Wakana Tang, Teodorica L. Bugawan, Omar Khwaja, Geert Jan Groeneveld, W. Birkhoff, Heidemarie Kletzl, Claire L Wood, Hanns Lochmüller, Sebastian Dziadek, Dirk Fischer, Christian Czech, Anne Marquet
المصدر: Neuromuscular disorders : NMD. 29(1)
مصطلحات موضوعية: 0301 basic medicine, Adult, Male, Adolescent, animal diseases, Administration, Oral, SMN1, Bioinformatics, spinal muscular atrophy, Muscular Atrophy, Spinal, 03 medical and health sciences, Exon, Young Adult, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, 0302 clinical medicine, Double-Blind Method, medicine, Humans, RNA, Messenger, Child, Genetics (clinical), business.industry, Alternative splicing, Infant, Survival of motor neuron, Spinal muscular atrophy, Motor neuron, Middle Aged, medicine.disease, SMA, Muscle atrophy, nervous system diseases, Survival of Motor Neuron 2 Protein, Alternative Splicing, 030104 developmental biology, medicine.anatomical_structure, Pyrimidines, nervous system, Neurology, Neuromuscular Agents, Child, Preschool, Pyrazines, Pediatrics, Perinatology and Child Health, Female, Neurology (clinical), medicine.symptom, business, 030217 neurology & neurosurgery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2cd3af212d2a80d491e04b1614b29a09Test
https://pubmed.ncbi.nlm.nih.gov/30553700Test -
3
المؤلفون: L Servais, Ksenija Gorni, Janbernd Kirschner, Y. Cleary, Eugenio Mercuri, W. Yeung, Marianne Gerber, Craig Campbell, Nathalie Goemans, Heidemarie Kletzl, Jeppe Buchbjerg, Omar Khwaja, M Pera, Christian Czech, Giovanni Baranello
المصدر: Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques. 46:S31
مصطلحات موضوعية: Oncology, medicine.medical_specialty, business.industry, Survival of motor neuron, General Medicine, SMN1, Spinal muscular atrophy, SMA, Placebo, medicine.disease, nervous system diseases, nervous system, Neurology, Tolerability, Pharmacokinetics, Internal medicine, medicine, Neurology (clinical), Adverse effect, business
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::9a18015a9b40a6ff28a3f3702bb2b47dTest
https://doi.org/10.1017/cjn.2019.163Test -
4
المؤلفون: John W. Day, Eugenio Mercuri, Nicolas Deconinck, L Servais, Heidemarie Kletzl, R. Masson, Marianne Gerber, Ksenija Gorni, Y. Cleary, C Cabalteja, Andrea Klein, T. Seabrook, M. El-Khairi, Basil T. Darras, Omar Khwaja, K. Gelblin, Christian Czech, Giovanni Baranello, C. Nguyen
المصدر: Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques. 46:S31
مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, business.industry, Survival of motor neuron, General Medicine, Spinal muscular atrophy, SMN1, 030105 genetics & heredity, SMA, Interim analysis, medicine.disease, Gastroenterology, Motor function, nervous system diseases, 03 medical and health sciences, 0302 clinical medicine, Neurology, Pharmacokinetics, Tolerability, Internal medicine, medicine, Neurology (clinical), business, 030217 neurology & neurosurgery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::193a4e5d38d8fcc2daf66d9496b47a16Test
https://doi.org/10.1017/cjn.2019.164Test -
5
المؤلفون: Baranello, Giovanni, Darras, Basil T, Day, John W, Deconinck, Nicolas, Klein, Andrea, Masson, Riccardo, Mercuri, Eugenio Maria, Rose, Kristy, El-Khairi, Muna, Gerber, Marianne, Gorni, Ksenija, Khwaja, Omar, Kletzl, Heidemarie, Scalco, Renata S, Seabrook, Timothy, Fontoura, Paulo, Servais, Laurent, FIREFISH Working Group: Joseph, J Volpe, John, Posner, Armin, Koch, Ulrich, Kellner, Rosaline, Quinlivan, Irina, Balikova, Patricia, Delbeke, Inge, Joniau, Valentine, Tahon, Sylvia, Wittevrongel, Elke De Vos, Edmar, Zanoteli, Rodrigo de Holanda Mendonça, Ciro Matsui Jr, Ana Letícia Fornazieri Darcie, Cleide, Machado, Maria Kiyoko Oyamada, Daniel de Souza Costa, Joyce, Martini, Graziela, Polido, Juliana Rodrigues Iannicelli, Juliana Caires de Oliveira Achili Ferreira, Wang, Yi, Chaoping, Hu, Yiyun, Shi, Shuizhen, Zhou, Xiaomei, Zhu, Chen, Qian, Shen, Li, Ying, Xiao, Zhenxuan, Zhou, Hui, Li, Sujuan, Wang, Hui, Xiong, Tian, Sang, Cuijie, Wei, Jing, Wen, Yiwen, Cao, Wenzhu, Li, Lun, Qin, Nina, Barisic, Ivan, Celovec, Martina Galiot Delic, Petra Kristina Ivkić, Nenad, Vukojević, Ivana, Kern, Boris, Najdanovic, Marin, Skugor, Odile, Boespflug-Tanguy, Teresa, Gidaro, Andrea, Seferian, Emmanuel, Barreau, Elodie Da Cunha, Céline, Lambotin, Nabila, Mnafek, Helene, Peche, Stephanie, Gilabert, Allison, Grange, Charlotte, Lilien, Darko, Milascevic, Ariadna, Perticari, Shotaro, Tachibana, Emanuela, Pagliano, Bianchi Marzoli, Stefania, Diletta, Santarsiero, Myriam Garcia Sierra, Gemma, Tremolada, Maria Teresa Arnoldi, Marta, Vigano, Riccardo, Zanin, Bissoli, Claudio Bruno, Noemi, Brolatti, Marina, Pedemonte, Enrico, Priolo, Giuseppe, Rao, Enrica, Spaletra, Lorenza, Sposetti, Elisa, Tassara, Simone, Morando, Paola, Tacchetti, Giacomo Pietro Comi, Alessandra, Govoni, Silvia Gabriella Osnaghi, Valeria, Minorini, Francesca, Abbati, Federica, Fassini, Michaela, Foa, Amalia, Lopopolo, Elisa, Minuti, Pane, Marika, Concetta, Palermo, Pera, Maria Carmela, Amorelli, Giulia Maria, Costanza, Barresi, Gugliemo, D'Amico, Orazi, Lorenzo, Coratti, Giorgia, De Sanctis, Roberto, Yasuhiro, Takeshima, Fumi, Gomi, Naoki, Kimura, Takanobu, Morimatsu, Mana, Okamoto, Toru, Furukawa, Maria, Mazurkiewicz-Bełdzińska, Mateusz, Koberda, Natalia, Kubiak, Urszula, Stodolska-Koberda, Agnieszka, Waśkowska, Jagoda, Kolendo, Agnieszka, Sobierajska-Rek, Dmitry, Vlodavets, Svetlana, Artemyeva, Evgenia, Melnik, Natalya, Leppenen, Nataliya, Yupatova, Elena, Litvinova, Anastasya, Monakhova, Yulia, Papina, Olga, Shidlovsckaia, Cornelia, Enzmann, Elea, Galiart, Konstantin, Gugleta, Patricia, Siems, Verena, Kreiliger, Christine Wondrusch Haschke, Haluk, Topaloglu, Ibrahim, Oncel, Didem, Ardicli, Nesibe Eroglu Ertugrul, Hizal, Gharibzadeh, Ceren, Gunbey, Bahadir, Konuskan, Selen Serel Arslan, Elams Ebru Yalcin, Fatma Gokcem Yildiz Sarikaya, Bora, Eldem, Sibel, Kadayıfçılar, Ipek, Alemdaroglu, Aynur Ayse Karaduman, Oznur Tunca Yilmaz, Lucia, Ambrosio, Anne, Fulton, Anna Maria Baglieri, Courtney, Dias, Elizabeth, Maczek, Elizabeth, Mirek, Amy, Pasternak, Shannon, Beres, Tina, Duong, Richard, Gee, Sally, Young
المساهمون: Giovanni, Baranello, Basil T, Darra, John W, Day, Nicolas, Deconinck, Andrea, Klein, Riccardo, Masson, Eugenio, Mercuri, Kristy, Rose, Muna, El-Khairi, Marianne, Gerber, Ksenija, Gorni, Omar, Khwaja, Heidemarie, Kletzl, Renata S, Scalco, Timothy, Seabrook, Paulo, Fontoura, Laurent, Servai, Ambrosio, Lucia
مصطلحات موضوعية: Male, Oral, Neuromuscular disease, RNA Splicing, Administration, Oral, 030204 cardiovascular system & hematology, Spinal Muscular Atrophies of Childhood, Bioinformatics, Dose-Response Relationship, 03 medical and health sciences, 0302 clinical medicine, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, medicine, Humans, Risdiplam, Type 1 Spinal Muscular Atrophy, 030212 general & internal medicine, Progression-free survival, 610 Medicine & health, Respiratory Tract Infections, Dose-Response Relationship, Drug, business.industry, Infant, Survival of motor neuron, General Medicine, Spinal muscular atrophy, medicine.disease, Survival of Motor Neuron 1 Protein, Progression-Free Survival, Clinical trial, Dose–response relationship, Settore MED/26 - NEUROLOGIA, Pyrimidines, nervous system, Neuromuscular Agents, RNA splicing, Administration, Female, Drug, business, Respiratory Insufficiency, Azo Compounds
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a876d0bc0f1f22a3373d8db456f23e0bTest
https://www.bib.irb.hr/1192690Test