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1
المؤلفون: Craig M McDonald, Francesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin, Heather Gordish-Dressman, Lauren Morgenroth, Christian Werner, James Li, Richard Able, Panayiota Trifillis, Már Tulinius, M Ryan, K Jones, N Goemans, C Campbell, JK Mah, K Selby, B Chabrol, Y Pereon, T Voit, T Gidaro, U Schara, JB Kirschner, Y Nevo, GP Comi, E Bertini, E Mercuri, J Colomer, A Nascimento, JJ Vilchez, M Tulinius, T Sejersen, F Muntoni, K Bushby, M Guglieri
المصدر: Journal of Comparative Effectiveness Research
مصطلحات موضوعية: medicine.medical_specialty, Vital capacity, nonsense mutation Duchenne muscular dystrophy, Duchenne muscular dystrophy, Nonsense mutation, efficacy, Walking, dystrophin, chemistry.chemical_compound, respiratory function, Internal medicine, loss of ambulation, medicine, Humans, Respiratory function, Respiratory system, Oxadiazoles, business.industry, Health Policy, ataluren, medicine.disease, Ataluren, Muscular Dystrophy, Duchenne, chemistry, Codon, Nonsense, Propensity score matching, Ambulatory, Study 019, business, Research Article
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cbb3f1627dca413773730a508656ed46Test
https://pubmed.ncbi.nlm.nih.gov/34791888Test -
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المؤلفون: C. Chiriboga, C. Bruno, T. Duong, D. Fischer, J. Kirschner, M. Scoto, E. Mercuri, M. Gerber, K. Gorni, H. Kletzl, I. Carruthers, C. Martin, T. Gidaro, F. Muntoni
المصدر: Neuromuscular Disorders. 32:S88
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::57d8f1005ecf05985d9dca368e4472cfTest
https://doi.org/10.1016/j.nmd.2022.07.195Test -
3
المؤلفون: C. Lilien, H. Reyngoudt, A. Seferian, T. Gidaro, M. Annoussamy, V. Chê, V. Decostre, I. Ledoux, J. Le Louër, E. Guemas, F. Muntoni, J. Hogrel, P. Carlier, L. Servais
المصدر: Neuromuscular Disorders. 31:S49
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::08944f00971d7f18cd89d72d40536406Test
https://doi.org/10.1016/j.nmd.2021.07.024Test -
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المؤلفون: J. Hogrel, V. Decostre, I. Ledoux, M. de Antonio, E. Niks, I. de Groot, V. Straub, F. Muntoni, V. Ricotti, T. Voit, A. Seferian, T. Gidaro, L. Servais
المصدر: Neuromuscular Disorders. 30:S84
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::09f53683028150dbe0816e8fb97089abTest
https://doi.org/10.1016/j.nmd.2020.08.132Test -
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المؤلفون: L. Servais, T. Gidaro, A. Seferian, E. Gasnier, A. Daron, A. Ulinici, M. Annoussamy, M. Grelet, D. Vissiere
المصدر: Neuromuscular Disorders. 29:S104
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::efeeeeb929601ef1f787d5207756c022Test
https://doi.org/10.1016/j.nmd.2019.06.244Test -
6
المؤلفون: M. Annoussamy, C. Lilien, T. Gidaro, V. Chê, U. Schara, A. D'Amico, J. Dowling, B. Darras, A. Daron, M. Mayer, A. Hernandez, C. Vuillerot, S. Fontaine, C. deLattre, R. Bellance, V. Biancalana, A. Buj-Bello, JR. Hogrel, H. Landy, L. Servais, null NatHis-MTM Study group
المساهمون: Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Saclay-Généthon, Généthon
المصدر: Neuromuscular Disorders
Neuromuscular Disorders, Elsevier, 2018, 28, pp.S71. ⟨10.1016/J.NMD.2018.06.168⟩
Neuromuscular Disorders, 2018, 28, pp.S71. ⟨10.1016/J.NMD.2018.06.168⟩مصطلحات موضوعية: 0303 health sciences, 03 medical and health sciences, 0302 clinical medicine, Neurology, Pediatrics, Perinatology and Child Health, Medizin, [SDV.BBM.BM]Life Sciences [q-bio]/Biochemistry, Molecular Biology/Molecular biology, Neurology (clinical), 030217 neurology & neurosurgery, Genetics (clinical), ComputingMilieux_MISCELLANEOUS, 030304 developmental biology
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::523b160f6e6af47bb5b87901ed2554deTest
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المؤلفون: Ulrike Schara, Diana Castro, T. Gidaro, S. Lutz, Ksenija Gorni, Janbernd Kirschner, Antonella Taglia, V. Doppler, F. Knipp, Christian Rummey, Jan J.G.M. Verschuuren, Thomas Meier, Gihan Tennekoon, J.J. Vílchez Padilla, E. Falcier, Luisa Politano, Craig M. McDonald, Chiara S. M. Straathof, S. Borell, S. Coopman, Matthew J. Will, Pierre-Yves Jeannet, Nathalie Goemans, Paolo Spagnolo, Jean-Marie Cuisset, Carsten G. Bönnemann, R.C. Richardson, G. Bernert, Thomas Sejersen, N. Muelas Gómez, Maria Grazia D'Angelo, Erika Brighina, Thomas Voit, S. Gandossini, Mika Leinonen, Susan T. Iannaccone, Oscar H. Mayer, Gunnar M. Buyse, Clemens Bloetzer, M. van den Hauwe, Susan D. Apkon, Richard S. Finkel, G. Buyse, P. D’Ambrosio, Erik K Henricson, Nanette C. Joyce, M. Hovmöller
المساهمون: Van den Hauwe, M, Meier, Thoma, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, Mayer, Oscar H., Buyse, Gunnar M., Bernert, G., Knipp, F., Buyse, G. M., Goemans, N., Van den Hauwe, M., Voit, T., Doppler, V., Gidaro, T., Cuisset, J. -. M., Coopman, S., Schara, U., Lutz, S., Kirschner, J., Borell, S., Will, M., D'Angelo, M. G., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D'Ambrosio, P., Taglia, A., Verschuuren, J. J. G. M., Straathof, C. S. M., VÃlchez Padilla, J. J., Muelas Gómez, N., Sejersen, T., Hovmã¶ller, M., Jeannet, P. -. Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Finkel, R., Bã¶nnemann, C., Mcdonald, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R. C.
المصدر: Europe PubMed Central
Neuromuscular Disorders, 27(4), 307-314مصطلحات موضوعية: Duchenne muscular dystrophy, medicine.medical_specialty, Vital capacity, Adolescent, peak expiratory flow, Respiratory Tract Diseases, Medizin, Clinical Neurology, Pulmonary insufficiency, Placebo group, Pediatrics, Pulmonary function testing, 03 medical and health sciences, FEV1/FVC ratio, 0302 clinical medicine, forced vital capacity, Internal medicine, medicine, Humans, In patient, Genetics(clinical), Pediatrics, Perinatology, and Child Health, Longitudinal Studies, Muscular Dystrophy, Child, Genetics (clinical), Old patients, business.industry, pulmonary function, respiratory system, Perinatology and Child Health, medicine.disease, Duchenne, Cross-Sectional Studies, Muscular Dystrophy, Duchenne, Respiratory Function Tests, Pediatrics, Perinatology and Child Health, Neurology, Neurology (clinical), Surgery, respiratory tract diseases, 030228 respiratory system, Cardiology, business, 030217 neurology & neurosurgery, circulatory and respiratory physiology
وصف الملف: Print-Electronic; application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b21864040305a8729c1e8de925e0e87cTest
http://hdl.handle.net/11577/3276306Test -
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المؤلفون: J. Hogrel, M. Annoussamy, A. Chabanon, A. Daron, Y. Péréon, C. Cancès, C. Vuillerot, N. Goemans, J. Cuisset, V. Laugel, U. Schara, E. Gargaun, T. Gidaro, A. Seferian, S. Turk, R. Hermosilla, E. Fournier, P. Baudin, P. Carlier, L. Servais, NatHis SMA Study Group
مصطلحات موضوعية: Electrophysiology, Nuclear magnetic resonance, Neurology, Chemistry, Pediatrics, Perinatology and Child Health, Medizin, Neurology (clinical), Function (mathematics), SMA, Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::56ebfbcdf83caebf37fa06b304e3d22eTest
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المؤلفون: J. Hogrel, M. Annoussamy, A. Chabanon, A. Daron, Y. Péréon, C. Cances, C. Vuillerot, N. Goemans, J. Cuisset, V. Laugel, U. Schara, E. Gargaun, T. Gidaro, A. Seferian, S. Turk, R. Hermosilla, E. Fournier, P. Baudin, P. Carlier, L. Servais, null Study Group
المصدر: Neuromuscular Disorders. 28:S56
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::e21334a59767ac7815086d2c2017c823Test
https://doi.org/10.1016/j.nmd.2018.06.116Test -
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المؤلفون: K. Aragon-Gawinska, A. Seferian, L. Vanden Brande, A. Daron, A. Ulinici, N. Deconinck, M. Annoussamy, C. Vuillerot, C. Cances, J. Ropars, M. Chouchane, Z. Balintova, S. Modrzejewska, I. Cuppen, I. Hughes, M. Illingworth, C. Marini-Bettolo, K. White, M. Scoto, T. Gidaro, L. Servais
المصدر: Neuromuscular Disorders. 28:S79
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::88328648b66bcc91ec5a16efe9214391Test
https://doi.org/10.1016/j.nmd.2018.06.196Test