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: Katharine, Bushby, Richard, Finkel, Brenda, Wong, Richard, Barohn, Craig, Campbell, Giacomo P, Comi, Anne M, Connolly, John W, Day, Kevin M, Flanigan, Nathalie, Goemans, Kristi J, Jones, Eugenio, Mercuri, Ros, Quinlivan, James B, Renfroe, Barry, Russman, Monique M, Ryan, Mar, Tulinius, Thomas, Voit, Steven A, Moore, H, Lee Sweeney, Richard T, Abresch, Kim L, Coleman, Michelle, Eagle, Julaine, Florence, Eduard, Gappmaier, Allan M, Glanzman, Erik, Henricson, Jay, Barth, Gary L, Elfring, Allen, Reha, Robert J, Spiegel, Michael W, O'donnell, Stuart W, Peltz, Craig M, Mcdonald, Kathleen, Ollendick

المصدر: Paediatrics Publications
Muscle & Nerve

مصطلحات موضوعية: Male, Pediatrics, Time Factors, Physiology, International Cooperation, Duchenne muscular dystrophy, nonsense mutation, Walking, Dystrophin, Orphan, chemistry.chemical_compound, 0302 clinical medicine, Outcome Assessment, Health Care, Clinical endpoint, Muscular Dystrophy, Prospective Studies, Child, Prospective cohort study, Pediatric, Oxadiazoles, 0303 health sciences, Nonsense mutation, 3. Good health, Codon, Nonsense, Child, Preschool, Drug, medicine.medical_specialty, Adolescent, Muscle disorder, Eteplirsen, Placebo, Dose-Response Relationship, Outcome Assessment (Health Care), 03 medical and health sciences, Cellular and Molecular Neuroscience, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Double-Blind Method, Genetic, Physiology (medical), medicine, Humans, Preschool, Codon, 030304 developmental biology, Dose-Response Relationship, Drug, business.industry, Main Articles, Duchenne, medicine.disease, orphan, Ataluren, Muscular Dystrophy, Duchenne, pediatric, Nonsense, chemistry, Neurology (clinical), genetic, business, 030217 neurology & neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0578329d6b528d6b608a1d76377c5da7Test
https://doi.org/10.1002/mus.24332Test

المؤلفون: Gunnar M. Buyse, Marleen Van den Hauwe, Thomas Meier, Nathalie Goemans

المصدر: Pediatric Pulmonology. 48:912-920

مصطلحات موضوعية: Pulmonary and Respiratory Medicine, medicine.medical_specialty, education.field_of_study, Vital capacity, business.industry, Duchenne muscular dystrophy, Population, medicine.disease, Placebo, Surgery, FEV1/FVC ratio, Internal medicine, Concomitant, Pediatrics, Perinatology and Child Health, medicine, Cardiology, Idebenone, Respiratory function, business, education, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::49968df2208de4a9df7347f998cba24fTest
https://doi.org/10.1002/ppul.22688Test