يعرض 1 - 2 نتائج من 2 نتيجة بحث عن '"Nathalie Goemans"', وقت الاستعلام: 1.04s تنقيح النتائج
  1. 1
    Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

    المؤلفون: Linda Lowes, Navid Khan, Lindsay N. Alfano, Helen Eliopoulos, Nathalie Goemans, Eugenio Mercuri, Craig M. McDonald, Jerry R. Mendell, Nanshi Sha

    المصدر: Journal of Neuromuscular Diseases

    مصطلحات موضوعية: Research Report, Duchenne muscular dystrophy, Male, medicine.medical_specialty, BOYS, Neuromuscular disease, Adolescent, Clinical Neurology, Walk Test, Walking, Eteplirsen, dystrophin, Morpholinos, Internal medicine, loss of ambulation, END-POINTS, medicine, Humans, In patient, eteplirsen, Child, POPULATION, Retrospective Studies, Science & Technology, 6-minute walk test, biology, business.industry, Neurosciences, Exons, medicine.disease, Duchenne, Exon skipping, PREVALENCE, Natural history, Muscular Dystrophy, Duchenne, Neurology, Case-Control Studies, Ambulatory, Mutation, biology.protein, Neurology (clinical), Neurosciences & Neurology, MUSCLE DISEASE, Dystrophin, business, Life Sciences & Biomedicine, EXONDYS 51

    وصف الملف: Print

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::92a2b68443493fe625b361d1d42ed491Test
    http://europepmc.org/articles/PMC8385516Test


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  2. 2
    A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment

    المؤلفون: Barry J. Byrne, Olga Mitelman, Sourav Santra, Jerry R. Mendell, Ashish Dugar, Anne M. Connolly, Hoda Z. Abdel-Hamid, Nathalie Goemans, Eugenio Mercuri, Peter Heydemann, Kathryn R. Wagner, Craig M. McDonald, Crystal Proud, Perry B. Shieh, James Signorovitch

    المصدر: Journal of neuromuscular diseases. 9(1)

    مصطلحات موضوعية: Duchenne muscular dystrophy, Male, Vital capacity, medicine.medical_specialty, Time Factors, 6-minutewalk test, Adolescent, Vital Capacity, Clinical Neurology, Walk Test, Eteplirsen, dystrophin, Pulmonary function testing, Morpholinos, DUCHENNE MUSCULAR-DYSTROPHY, 6-MINUTE WALK TEST, FEV1/FVC ratio, DRISAPERSEN, forced vital capacity, loss of ambulation, Internal medicine, END-POINTS, Outcome Assessment, Health Care, Medicine, Humans, eteplirsen, Prospective Studies, Registries, Mobility Limitation, Child, Retrospective Studies, Science & Technology, business.industry, Proportional hazards model, DISEASE PROGRESSION, Neurosciences, Repeated measures design, NATURAL-HISTORY, Muscular Dystrophy, Duchenne, Neurology, Ambulatory, Disease Progression, TRIAL, Neurosciences & Neurology, Neurology (clinical), business, Life Sciences & Biomedicine, Natural history study

    وصف الملف: Print

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::00a25355a896eeef4db17d9cfa0c69a6Test
    https://pubmed.ncbi.nlm.nih.gov/34420980Test


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