المؤلفون: Nathalie Goemans, Eugenio Mercuri, Elena Belousova, Hirofumi Komaki, Alberto Dubrovsky, Craig M. McDonald, John E. Kraus, Afrodite Lourbakos, Zhengning Lin, Giles Campion, Susanne X. Wang, Craig Campbell, A. Araujo, E. Bertini, P. Born, C. Cances, B. Chabrol, J.-H. Chae, J. Colomer Oferil, G.P. Comi, J.-M. Cuisset, G. D'Anjou, I. Desguerre, R. Erazo Torricelli, R. Escobar, D. Feder, A. Ferlini, R. Giugliani, E. Henricson, A. Herczegfalvi, Y.-J. Jong, S. Kimura, J.-B. Kirschner, K. Kleinsteuber, A. Kostera-Pruszczyk, M. Kudr, W. Mueller-Felber, E.H. Niks, K. Ogata, C. Palermo, M. Pane, I. Pascual, Y. Pereon, S. Raskin, M. Rasmussen, U. Reed, U. Schara, K. Selby, C. Sobreira, Y. Takeshima, J.J. Vilchez Padilla, G. Vita, P. Vondracek, G. Wiegand, E. Wilichowski

المساهمون: Schara, Ulrike (Beitragende*r)

المصدر: Paediatrics Publications
Neuromuscular Disorders
Neuromuscular Disorders, 28(1), 4-15

مصطلحات موضوعية: 0301 basic medicine, Duchenne muscular dystrophy, Male, medicine.medical_specialty, Adolescent, Population, six-minute walking distance, Medizin, Oligonucleotides, Phases of clinical research, Socio-culturale, Motor Activity, Placebo, Pediatrics, Dystrophin, 03 medical and health sciences, 0302 clinical medicine, Double-Blind Method, Internal medicine, Statistical significance, Medicine, Humans, Antisense oligonucleotide, education, Adverse effect, Child, Drisapersen, Genetics (clinical), education.field_of_study, business.industry, Perinatology and Child Health, medicine.disease, Muscular Dystrophy, Duchenne, 030104 developmental biology, Treatment Outcome, Neurology, Child, Preschool, Pediatrics, Perinatology and Child Health, Ambulatory, Neurology (clinical), 6-minute walking distance, antisense oligonucleotide, drisapersen, dystrophin, exon skipping, business, 030217 neurology & neurosurgery, Exon skipping, Biomarkers, Six-minute walking distance

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2b23f19cae216fd0ab3f5c58c553a0a7Test
https://pubmed.ncbi.nlm.nih.gov/29203355Test

المؤلفون: Gunnar M. Buyse, Imelda J. M. de Groot, Daisy Thijs, Berten Ceulemans, Ulrike Schara, Marleen Van den Hauwe, Nathalie Goemans, Thomas Meier, Luc Mertens

المصدر: Neuromuscular disorders
Neuromuscular Disorders, 21, 396-405
Neuromuscular Disorders, 21, 6, pp. 396-405

مصطلحات موضوعية: Male, musculoskeletal diseases, medicine.medical_specialty, Human Movement & Fatigue DCN 1: Perception and Action [NCEBP 10], Adolescent, Ubiquinone, Duchenne muscular dystrophy, Vital Capacity, Placebo-controlled study, Medizin, Placebo, Antioxidants, law.invention, Randomized controlled trial, Double-Blind Method, law, Forced Expiratory Volume, Natriuretic Peptide, Brain, medicine, Idebenone, Humans, Respiratory function, Longitudinal Studies, Muscle Strength, Child, Genetics (clinical), Dose-Response Relationship, Drug, business.industry, Troponin I, medicine.disease, Surgery, Clinical trial, Muscular Dystrophy, Duchenne, Treatment Outcome, Neurology, Tolerability, Anesthesia, Pediatrics, Perinatology and Child Health, Disease Progression, Human Movement & Fatigue Perception and Action [NCEBP 10], Neurology (clinical), Human medicine, business, medicine.drug

وصف الملف: pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::58169882307d3fa2793715c30e602010Test
https://hdl.handle.net/2066/95981Test

المؤلفون: James Signorovitch, S. Ward, Nathalie Goemans, Eugenio Mercuri, Erin McDonnell, Zhiwen Yao, Gautam Sajeev

المصدر: Neuromuscular Disorders. 27:S232

مصطلحات موضوعية: SIX MINUTE WALK, medicine.medical_specialty, business.industry, Duchenne muscular dystrophy, medicine.disease, Placebo, Clinical trial, Natural history, Physical medicine and rehabilitation, Neurology, Consistency (statistics), Pediatrics, Perinatology and Child Health, medicine, Physical therapy, In patient, Neurology (clinical), business, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::a5bc1e86978f0c6ec8f9a8e3cca9f398Test
https://doi.org/10.1016/j.nmd.2017.06.493Test

المؤلفون: C. Wardell, Nathalie Goemans, Eugenio Mercuri, T. Voit, G. Campion, Craig M. McDonald, R. Wilson

المصدر: Neuromuscular Disorders. 24:829-830

مصطلحات موضوعية: Pediatrics, medicine.medical_specialty, business.industry, Repeated measures design, Placebo, Confidence interval, Prolonged exposure, Walking distance, Standard error, Neurology, Pediatrics, Perinatology and Child Health, Ambulatory, Medicine, Population study, Neurology (clinical), business, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::f1f94a957fd5c86d92c2d3dd5bad4a30Test
https://doi.org/10.1016/j.nmd.2014.06.129Test

المؤلفون: E. Bertini, Anna Lusakowska, L. H. van den Berg, W. Hauke, Bruno Scherrer, P. Berna, C. André, Janbernd Kirschner, Nathalie Goemans, Eugenio Mercuri, Gianluca Vita, M. Mayer, Brigitte Estournet, Brigitte Chabrol, M. Walter, R. Pruss, S. Fontaine-Carbonel, Ulrike Schara, Ksenija Gorni, Claudio Bruno, Wolfgang Müller-Felber, Francesco Muntoni, Nicolas Deconinck, H. Roper, François Rivier, G. Comi, Jean-Marie Cuisset, Eric Dessaud, Hanns Lochmüller, V. Cuvier

المصدر: Neuromuscular Disorders. 24:920-921

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Spinal muscular atrophy, medicine.disease, Placebo, SMA, Surgery, Pulmonary function testing, Clinical trial, chemistry.chemical_compound, Neurology, chemistry, Anesthesia, Pediatrics, Perinatology and Child Health, medicine, Clinical endpoint, Olesoxime, Neurology (clinical), Adverse effect, business, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::64e01fd40621b2987e5b259c21980ef2Test
https://doi.org/10.1016/j.nmd.2014.06.418Test

المؤلفون: T. Voit, C. Wardell, Nathalie Goemans, G. Campion, Eugenio Mercuri, R. Wilson, C. McDonald

المصدر: Neuromuscular Disorders. 24:922

مصطلحات موضوعية: Moderate to severe, medicine.medical_specialty, education.field_of_study, Proteinuria, business.industry, Disease progression, Population, Phases of clinical research, Placebo, Gastroenterology, Surgery, Walking distance, Neurology, Internal medicine, Pediatrics, Perinatology and Child Health, Medicine, Neurology (clinical), medicine.symptom, business, education, Mild disease, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::b5385ac1d904ef9910e18ec3d2d93465Test
https://doi.org/10.1016/j.nmd.2014.06.422Test

المؤلفون: Craig M. McDonald, R. Wilson, T. Voit, G. Campion, Nathalie Goemans, Eugenio Mercuri, C. Wardell

المصدر: Neuromuscular Disorders. 24:829

مصطلحات موضوعية: medicine.medical_specialty, education.field_of_study, business.industry, Beta-2 microglobulin, Gross motor skill, Population, Phases of clinical research, Placebo, Neurology, Disease severity, Internal medicine, Pediatrics, Perinatology and Child Health, Ambulatory, Clinical endpoint, Physical therapy, Medicine, Neurology (clinical), business, education, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::088faa28aa42a8982ec6c799ae7d1cb2Test
https://doi.org/10.1016/j.nmd.2014.06.128Test

المؤلفون: Hermien E. Kan, G. Campion, Erik H. Niks, B.H.A. Wokke, Nathalie Goemans, J.J.G. Verschuuren, Melissa T. Hooijmans

المصدر: Neuromuscular Disorders. 24:852

مصطلحات موضوعية: Muscle tissue, medicine.medical_specialty, business.industry, Duchenne muscular dystrophy, Urology, medicine.disease, Placebo, Exon skipping, Surgery, medicine.anatomical_structure, Neurology, Tibialis anterior muscle, Pediatrics, Perinatology and Child Health, medicine, Clinical endpoint, Biomarker (medicine), Neurology (clinical), business, Genetics (clinical), Drisapersen

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::a4191817da722bd72af1ea7da116a235Test
https://doi.org/10.1016/j.nmd.2014.06.199Test

المؤلفون: Nathalie Goemans, A. Morgan, F. Lawrence, R. Wilson, Eugenio Mercuri, L. Callendret, G. Campion, M. Eagle

المصدر: Neuromuscular Disorders. 23:773

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Duchenne muscular dystrophy, Cardiorespiratory fitness, Placebo, medicine.disease, Pulmonary function testing, Clinical trial, Neurology, Quality of life, Concomitant, Pediatrics, Perinatology and Child Health, Physical therapy, Medicine, Neurology (clinical), business, Genetics (clinical), Natural history study

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::69a561f9d9f7039f46b7224116b3acbfTest
https://doi.org/10.1016/j.nmd.2013.06.482Test

المؤلفون: Stuart W. Peltz, Gary Elfring, K. Bushby, J. Barth, A. Reha, Craig M. McDonald, Eugenio Mercuri, Nathalie Goemans, Basil T. Darras, Francesco Muntoni

المصدر: Neuromuscular Disorders. 21:651

مصطلحات موضوعية: Pediatrics, medicine.medical_specialty, Neurology, business.industry, Pediatrics, Perinatology and Child Health, Nonsense mutation, Disease progression, Outcome measures, Medicine, Neurology (clinical), Placebo, business, Genetics (clinical)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::31cac46a94695c6eba31ace942c82659Test
https://doi.org/10.1016/j.nmd.2011.06.793Test