المؤلفون: Nathalie Goemans, Brenda Wong, Marleen Van den Hauwe, James Signorovitch, Gautam Sajeev, David Cox, John Landry, Madeline Jenkins, Ibrahima Dieye, Zhiwen Yao, Intekhab Hossain, Susan J Ward, Collaborative Trajectory Analysis Project (cTAP)

المصدر: PLoS ONE, Vol 15, Iss 6, p e0232870 (2020)
PLoS ONE

مصطلحات موضوعية: 0301 basic medicine, Male, Heredity, Physiology, Genetic Linkage, Duchenne muscular dystrophy, Walking, Duchenne Muscular Dystrophy, 030105 genetics & heredity, Pediatrics, Muscular Dystrophies, DISEASE, law.invention, Tadalafil, Efficacy, 0302 clinical medicine, Randomized controlled trial, DESIGN, law, Medicine and Health Sciences, Medicine, Child, Multidisciplinary, Organic Compounds, Prognosis, Phase III clinical investigation, Multidisciplinary Sciences, Chemistry, Neurology, Neuromuscular Agents, X-Linked Traits, Sex Linkage, Ambulatory, Physical Sciences, Disease Progression, Science & Technology - Other Topics, Steroids, TRIAL, Research Article, medicine.medical_specialty, Drug Research and Development, Adolescent, Science, ATALUREN, Placebo, Research and Analysis Methods, 03 medical and health sciences, DRISAPERSEN, Diagnostic Medicine, Genetics, Humans, Clinical Trials, Computer Simulation, Clinical Genetics, Pharmacology, Science & Technology, business.industry, Biological Locomotion, Clinical study design, Organic Chemistry, Chemical Compounds, Biology and Life Sciences, medicine.disease, Clinical trial, Muscular Dystrophy, Duchenne, Sample size determination, Sample Size, Physical therapy, Exercise Test, Clinical Medicine, business, 030217 neurology & neurosurgery, Follow-Up Studies

وصف الملف: Electronic-eCollection

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c05aa276ab26d60ebede754c9790ea90Test
https://lirias.kuleuven.be/handle/20.500.12942/704020Test

المؤلفون: Edward M. Kaye, K. Berry, Nathalie Goemans, Lindsay N. Alfano, Eugenio Mercuri, James Shao, Linda Lowes, Jerry R. Mendell

المصدر: Annals of Neurology. 79:257-271

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, business.industry, Duchenne muscular dystrophy, medicine.disease, Placebo, Eteplirsen, Exon skipping, Pulmonary function testing, Surgery, 03 medical and health sciences, 030104 developmental biology, 0302 clinical medicine, Neurology, Internal medicine, Ambulatory, medicine, Respiratory muscle, Neurology (clinical), Muscular dystrophy, business, 030217 neurology & neurosurgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::1e574246330f394154357efdef7a73caTest
https://doi.org/10.1002/ana.24555Test

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: 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

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