المؤلفون: Nicolas Deconinck, Steve S Hood, Haluk Topaloglu, Lia Liefaard, C. Wardell, Rosamund Wilson, Sjef J. de Kimpe, John E. Kraus, Michelle Eagle, Katie Rolfe, Laurent Servais, Joanna Nakielny, Afrodite Lourbakos, Michela Guglieri, Valeria Ricotti, Giles G Campion, Allison A Morgan, Francesco Muntoni, Volker Straub, Padraig Wright, Thomas Voit, Naashika Quarcoo

المصدر: The Lancet Neurology. 13:987-996

مصطلحات موضوعية: Male, medicine.medical_specialty, Duchenne muscular dystrophy, Population, Anti-Inflammatory Agents, Oligonucleotides, Walking, Real-Time Polymerase Chain Reaction, Eteplirsen, Placebo, law.invention, Dystrophin, Double-Blind Method, Randomized controlled trial, Adrenal Cortex Hormones, law, Internal medicine, Clinical endpoint, medicine, Humans, Child, education, Adverse effect, Drisapersen, education.field_of_study, business.industry, Exons, medicine.disease, Muscular Dystrophy, Duchenne, Treatment Outcome, Area Under Curve, Child, Preschool, Physical therapy, Female, Neurology (clinical), business, Gene Deletion

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4376b8b1e340ad1c94d88d59dcefb3a3
https://doi.org/10.1016/s1474-4422(14)70195-4

المؤلفون: Rider D; Silence Therapeutics GmbH, Berlin 13125, Germany., Chivers S; Integrated Biologix, Basel CH-4051, Switzerland., Aretz J; Silence Therapeutics GmbH, Berlin 13125, Germany., Eisermann M; Silence Therapeutics GmbH, Berlin 13125, Germany., Löffler K; Silence Therapeutics GmbH, Berlin 13125, Germany., Hauptmann J; Silence Therapeutics GmbH, Berlin 13125, Germany., Morrison E; Silence Therapeutics GmbH, Berlin 13125, Germany., Campion G; Silence Therapeutics PLC, London W14 8TH, UK.

المصدر: Toxicological sciences : an official journal of the Society of Toxicology [Toxicol Sci] 2022 Sep 24; Vol. 189 (2), pp. 237-249.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Oxford University Press Country of Publication: United States NLM ID: 9805461 Publication Model: Print Cited Medium: Internet ISSN: 1096-0929 (Electronic) Linking ISSN: 10960929 NLM ISO Abbreviation: Toxicol Sci Subsets: MEDLINE

مواضيع طبية MeSH: Acetylgalactosamine*/metabolism , Acetylgalactosamine*/toxicity , Cardiovascular Diseases*, Alanine Transaminase ; Alkaline Phosphatase ; Animals ; Cytokines ; Ethers ; Humans ; Lipoprotein(a) ; Macaca fascicularis ; RNA, Messenger ; RNA, Small Interfering/genetics ; RNA, Small Interfering/metabolism ; Rabbits ; Rats

المؤلفون: Rider DA; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany. Electronic address: d.rider@silence-therapeutics.com., Eisermann M; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Löffler K; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Aleku M; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Swerdlow DI; Silence Therapeutics PLC, 72 Hammersmith Road, London, W14 8TH, London, UK., Dames S; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Hauptmann J; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Morrison E; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Lindholm MW; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Schubert S; Silence Therapeutics GmbH, Robert-Rössle-Straße 10, 13125, Berlin, Germany., Campion G; Silence Therapeutics PLC, 72 Hammersmith Road, London, W14 8TH, London, UK.

المصدر: Atherosclerosis [Atherosclerosis] 2022 May; Vol. 349, pp. 240-247. Date of Electronic Publication: 2022 Apr 01.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Elsevier Country of Publication: Ireland NLM ID: 0242543 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-1484 (Electronic) Linking ISSN: 00219150 NLM ISO Abbreviation: Atherosclerosis Subsets: MEDLINE

مواضيع طبية MeSH: Cardiovascular Diseases*/drug therapy , Cardiovascular Diseases*/genetics , Hyperlipidemias*, Apolipoproteins A ; Apolipoproteins B ; Humans ; Lipoprotein(a) ; RNA, Messenger ; RNA, Small Interfering/genetics

المؤلفون: McDonald CM; University of California, Davis Sacramento California., Wong B; Cincinnati Children's Hospital Cincinnati Ohio., Flanigan KM; Nationwide Children's Hospital Columbus Ohio., Wilson R; Spica Consultants Ltd Marlborough United Kingdom., de Kimpe S; BioMarin Nederland B.V. Leiden The Netherlands., Lourbakos A; BioMarin Nederland B.V. Leiden The Netherlands., Lin Z; BioMarin Nederland B.V. Leiden The Netherlands., Campion G; BioMarin Nederland B.V. Leiden The Netherlands.

مؤلفون مشاركون: DEMAND V study group

المصدر: Annals of clinical and translational neurology [Ann Clin Transl Neurol] 2018 Jul 11; Vol. 5 (8), pp. 913-926. Date of Electronic Publication: 2018 Jul 11 (Print Publication: 2018).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Wiley Periodicals, Inc on behalf of American Neurological Association Country of Publication: United States NLM ID: 101623278 Publication Model: eCollection Cited Medium: Print ISSN: 2328-9503 (Print) Linking ISSN: 23289503 NLM ISO Abbreviation: Ann Clin Transl Neurol Subsets: PubMed not MEDLINE

المؤلفون: Goemans N; Department of Pediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium. Electronic address: nathalie.goemans@uzleuven.be., Mercuri E; Pediatric Neurology, Catholic University, Rome, Italy., Belousova E; Research and Clinical Institute of Pediatrics, Pirogov Russian National Research Medical University, Moscow, Russia., Komaki H; Department of Child Neurology, National Center of Neurology and Psychiatry, Tokyo, Japan., Dubrovsky A; Fundacion Cenit, Instituto de Neurociencias, Fundación Favaloro, Buenos Aires, Argentina., McDonald CM; School of Medicine, University of California, Davis, Sacramento, CA, USA., Kraus JE; GlaxoSmithKline, Research Triangle Park, NC, USA., Lourbakos A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Lin Z; BioMarin Pharmaceutical Inc., Novato, CA, USA., Campion G; BioMarin Pharmaceutical Inc., Novato, CA, USA., Wang SX; BioMarin Pharmaceutical Inc., Novato, CA, USA., Campbell C; Paediatric Neurology, Schulich School of Medicine, Western University, London, Canada.

مؤلفون مشاركون: DEMAND III study group

المصدر: Neuromuscular disorders : NMD [Neuromuscul Disord] 2018 Jan; Vol. 28 (1), pp. 4-15. Date of Electronic Publication: 2017 Dec 06.

نوع المنشور: Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE

مواضيع طبية MeSH: Muscular Dystrophy, Duchenne/*therapy , Oligonucleotides/*therapeutic use, Adolescent ; Biomarkers/blood ; Child ; Child, Preschool ; Double-Blind Method ; Humans ; Male ; Motor Activity ; Muscular Dystrophy, Duchenne/blood ; Oligonucleotides/adverse effects ; Oligonucleotides/metabolism ; Treatment Outcome

المؤلفون: Goemans N; Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium. Electronic address: nathalie.goemans@uzleuven.be., Tulinius M; The Queen Silvia Children's Hospital, Gothenburg, Sweden., Kroksmark AK; The Queen Silvia Children's Hospital, Gothenburg, Sweden., Wilson R; Spica Consultants Ltd, Marlborough, United Kingdom., van den Hauwe M; Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium., Campion G; BioMarin Nederland BV, The Netherlands.

المصدر: Neuromuscular disorders : NMD [Neuromuscul Disord] 2017 Mar; Vol. 27 (3), pp. 203-213. Date of Electronic Publication: 2016 Nov 25.

نوع المنشور: Comparative Study; Journal Article

بيانات الدورية: Publisher: Pergamon Press Country of Publication: England NLM ID: 9111470 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2364 (Electronic) Linking ISSN: 09608966 NLM ISO Abbreviation: Neuromuscul Disord Subsets: MEDLINE

مواضيع طبية MeSH: Disease Progression* , Exercise Test* , Walking*, Adrenal Cortex Hormones/*pharmacology , Muscular Dystrophy, Duchenne/*drug therapy , Oligonucleotides/*pharmacology, Adolescent ; Adrenal Cortex Hormones/administration & dosage ; Child ; Cohort Studies ; Humans ; Male ; Oligonucleotides/administration & dosage ; Outcome Assessment, Health Care

المؤلفون: Goemans NM; University Hospitals Leuven, Leuven, Belgium., Tulinius M; University of Gothenburg, Gothenburg, Sweden., van den Hauwe M; University Hospitals Leuven, Leuven, Belgium., Kroksmark AK; University of Gothenburg, Gothenburg, Sweden., Buyse G; University Hospitals Leuven, Leuven, Belgium., Wilson RJ; Spica Consultants Ltd, Marlborough, United Kingdom., van Deutekom JC; BioMarin Pharmaceutical Inc., Leiden, Netherlands., de Kimpe SJ; BioMarin Pharmaceutical Inc., Leiden, Netherlands., Lourbakos A; BioMarin Pharmaceutical Inc., Leiden, Netherlands., Campion G; BioMarin Pharmaceutical Inc., Leiden, Netherlands.

المصدر: PloS one [PLoS One] 2016 Sep 02; Vol. 11 (9), pp. e0161955. Date of Electronic Publication: 2016 Sep 02 (Print Publication: 2016).

نوع المنشور: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article

بيانات الدورية: Publisher: Public Library of Science Country of Publication: United States NLM ID: 101285081 Publication Model: eCollection Cited Medium: Internet ISSN: 1932-6203 (Electronic) Linking ISSN: 19326203 NLM ISO Abbreviation: PLoS One Subsets: MEDLINE

مواضيع طبية MeSH: Muscular Dystrophy, Duchenne/*drug therapy , Oligonucleotides/*therapeutic use, Adolescent ; Child ; Child, Preschool ; Dystrophin/genetics ; Dystrophin/metabolism ; Exercise Test ; Humans ; Male ; Muscle, Skeletal/metabolism ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/metabolism ; Oligonucleotides/adverse effects ; Oligonucleotides/pharmacokinetics ; Treatment Outcome ; Walking/physiology

المؤلفون: Straub V; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Balabanov P; European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Management Department, CNS and Ophthalmology Office, London, UK., Bushby K; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Ensini M; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Goemans N; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium., De Luca A; Unit of Pharmacology, Department of Pharmacy-Drug Sciences, University of Bari, Bari, Italy., Pereda A; Duchenne Parent Project, Madrid, Spain., Hemmings R; Medicines and Healthcare Product Regulatory Agency, London, UK., Campion G; BioMarin Pharmaceutical, Developmental Sciences, Novato, CA, USA., Kaye E; Sarepta Therapeutics, Cambridge, MA, USA., Arechavala-Gomeza V; Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Goyenvalle A; Université de Versailles Saint Quentin en Yvelines, Inserm U1179, Montigny le Bretonneux, France., Niks E; Leiden University Medical Center, Leiden, Netherlands., Veldhuizen O; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Furlong P; Parent Project Muscular Dystrophy, Hackensack, NJ, USA., Stoyanova-Beninska V; Medicines Evaluation Board, Utrecht, Netherlands., Wood MJ; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK., Johnson A; Joining Jack, Wigan, UK., Mercuri E; Pediatric Neurology, Catholic University, Rome, Italy., Muntoni F; Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, University College London Institute of Child Health and Great Ormond Street Hospital, London, UK., Sepodes B; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal., Haas M; European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Management Department, CNS and Ophthalmology Office, London, UK., Vroom E; United Parent Projects Muscular Dystrophy, Amsterdam, Netherlands., Aartsma-Rus A; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK; Leiden University Medical Center, Leiden, Netherlands. Electronic address: a.m.rus@lumc.nl.

المصدر: The Lancet. Neurology [Lancet Neurol] 2016 Jul; Vol. 15 (8), pp. 882-890.

نوع المنشور: Journal Article; Review

بيانات الدورية: Publisher: Lancet Pub. Group Country of Publication: England NLM ID: 101139309 Publication Model: Print Cited Medium: Internet ISSN: 1474-4465 (Electronic) Linking ISSN: 14744422 NLM ISO Abbreviation: Lancet Neurol Subsets: MEDLINE

مواضيع طبية MeSH: Orphan Drug Production* , Public-Private Sector Partnerships*, Muscular Dystrophy, Duchenne/*therapy, Drug Approval ; Genetic Therapy ; Humans ; Muscular Dystrophy, Duchenne/genetics ; Outcome Assessment, Health Care

المؤلفون: Anthony K; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Arechavala-Gomeza V; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Taylor LE; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Vulin A; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Kaminoh Y; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Torelli S; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Feng L; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Janghra N; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Bonne G; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Beuvin M; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Barresi R; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Henderson M; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Laval S; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Lourbakos A; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Campion G; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Straub V; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Voit T; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Sewry CA; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Morgan JE; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Flanigan KM; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Muntoni F; From The Dubowitz Neuromuscular Centre (K.A., V.A.-G., S.T., L.F., N.J., C.A.S., J.E.M., F.M.), UCL, Institute of Child Health, London, UK; The Center for Gene Therapy (L.E.T., A.V., Y.K., K.M.F.), The Research Institute at Nationwide Children's Hospital, Columbus, OH; Institut de Myologie (G.B., M.B., T.V.), UPMC UM76, INSERM U 794, CNRS UMR 7215, Paris, France; Institute of Genetic Medicine (R.B., M.H., S.L., V.S.), Newcastle University, UK; and Prosensa Therapeutics (A.L., G.C.), Leiden, the Netherlands. V.A.-G. is currently affiliated with the Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain. f.muntoni@ucl.ac.uk.

المصدر: Neurology [Neurology] 2014 Nov 25; Vol. 83 (22), pp. 2062-9. Date of Electronic Publication: 2014 Oct 29.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 0401060 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1526-632X (Electronic) Linking ISSN: 00283878 NLM ISO Abbreviation: Neurology Subsets: MEDLINE

مواضيع طبية MeSH: Dystrophin/*analysis , Translational Research, Biomedical/*standards, Dystrophin/genetics ; Humans ; Medical Laboratory Science/methods ; Medical Laboratory Science/standards ; Muscular Dystrophy, Duchenne/diagnosis ; Muscular Dystrophy, Duchenne/genetics ; Observer Variation ; Translational Research, Biomedical/methods

المؤلفون: Voit T; Institut de Myologie, Universite Pierre et Marie Curie, GH Pitié-Salpêtrière, UPMC-INSERM UMR 974, Paris, France. Electronic address: t.voit@institut-myologie.org., Topaloglu H; Department of Pediatrics, Hacettepe University, Ankara, Turkey., Straub V; Institute of Genetic Medicine, University of Newcastle, Newcastle, UK., Muntoni F; The Dubowitz Neuromuscular Centre, University College London Institute of Child Health, London, UK., Deconinck N; Department of Neurology, Universitair Ziekenhuis Gent, Ghent, Belgium; Department of Paediatric Neurology, Hopital des Enfants reine Fabiola, ULB, Belgium., Campion G; Prosensa Therapeutics BV, Leiden, Netherlands., De Kimpe SJ; Prosensa Therapeutics BV, Leiden, Netherlands., Eagle M; Institute of Genetic Medicine, University of Newcastle, Newcastle, UK., Guglieri M; Institute of Genetic Medicine, University of Newcastle, Newcastle, UK., Hood S; GlaxoSmithKline, London, UK., Liefaard L; GlaxoSmithKline, Stevenage, UK., Lourbakos A; Prosensa Therapeutics BV, Leiden, Netherlands., Morgan A; Prosensa Therapeutics BV, Leiden, Netherlands; Methis Clinical, Ascot, Berkshire, UK., Nakielny J; GlaxoSmithKline, London, UK., Quarcoo N; GlaxoSmithKline, London, UK., Ricotti V; The Dubowitz Neuromuscular Centre, University College London Institute of Child Health, London, UK., Rolfe K; GlaxoSmithKline, London, UK., Servais L; Institut de Myologie, Universite Pierre et Marie Curie, GH Pitié-Salpêtrière, UPMC-INSERM UMR 974, Paris, France., Wardell C; Prosensa Therapeutics BV, Leiden, Netherlands; GlaxoSmithKline, London, UK., Wilson R; Spica Consultants Ltd, Marlborough, UK., Wright P; GlaxoSmithKline, London, UK; Takeda, London, UK., Kraus JE; GlaxoSmithKline, Raleigh-Durham, NC, USA.

المصدر: The Lancet. Neurology [Lancet Neurol] 2014 Oct; Vol. 13 (10), pp. 987-96. Date of Electronic Publication: 2014 Sep 07.

نوع المنشور: Clinical Trial, Phase II; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Lancet Pub. Group Country of Publication: England NLM ID: 101139309 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1474-4465 (Electronic) Linking ISSN: 14744422 NLM ISO Abbreviation: Lancet Neurol Subsets: MEDLINE

مواضيع طبية MeSH: Muscular Dystrophy, Duchenne/*drug therapy , Oligonucleotides/*therapeutic use, Adrenal Cortex Hormones/therapeutic use ; Anti-Inflammatory Agents/therapeutic use ; Area Under Curve ; Child ; Child, Preschool ; Double-Blind Method ; Dystrophin/genetics ; Exons ; Female ; Gene Deletion ; Humans ; Male ; Muscular Dystrophy, Duchenne/genetics ; Oligonucleotides/adverse effects ; Real-Time Polymerase Chain Reaction ; Treatment Outcome ; Walking