-
1دورية أكاديمية
المؤلفون: Frank, Diane E., Schnell, Frederick J., Akana, Cody, El-Husayni, Saleh H., Desjardins, Cody A., Morgan, Jennifer, Charleston, Jay S., Sardone, Valentina, Domingos, Joana, Dickson, George, Straub, Volker, Guglieri, Michela, Mercuri, Eugenio, Servais, Laurent, Muntoni, Francesco
المصدر: Neurology, 94 (21), e2270-e2282 (2020-05-26)
مصطلحات موضوعية: Administration, Intravenous, Adolescent, Child, Dose-Response Relationship, Drug, Double-Blind Method, Dystrophin/biosynthesis/genetics, Fluorescent Antibody Technique, Humans, Male, Muscle, Skeletal/metabolism, Muscular Dystrophy, Duchenne/blood/drug therapy/genetics, Oligonucleotides/therapeutic use, Sequence Deletion/drug effects, Human health sciences, Pediatrics, Neurology, Sciences de la santé humaine, Pédiatrie, Neurologie
Relation: urn:issn:0028-3878; urn:issn:1526-632X
URL الوصول: https://orbi.uliege.be/handle/2268/256225
-
2دورية أكاديمية
لا يتم عرض هذه النتيجة على الضيوف.
تسجيل الدخول للوصول الكامل. -
3دورية أكاديمية
لا يتم عرض هذه النتيجة على الضيوف.
تسجيل الدخول للوصول الكامل. -
4دورية أكاديمية
لا يتم عرض هذه النتيجة على الضيوف.
تسجيل الدخول للوصول الكامل. -
5دورية أكاديمية
المؤلفون: Frank, Diane E., Carter, William G.
المصدر: Journal of Cell Science; 3/15/2004, Vol. 117 Issue 8, p1351-1363, 13p, 13 Color Photographs, 14 Black and White Photographs, 9 Diagrams, 1 Chart, 8 Graphs
مصطلحات موضوعية: KERATINOCYTES, EPIDERMIS, INTEGRINS, CELL migration, CELL adhesion, EXTRACELLULAR matrix
-
6دورية
المؤلفون: Tsodikov, Oleg V., Saecker, Ruth M., Melcher, Sonya E., Levandoski, Mark M., Frank, Diane E., Capp, Michael W., Record, M.Thomas
المصدر: JMB Online (Journal of Molecular Biology); December 1999, Vol. 294 Issue: 3 p639-655, 17p
-
7دورية
المؤلفون: Levandoski, Mark M., Tsodikov, Oleg V., Frank, Diane E., Melcher, Sonya E., Saecker, Ruth M., Jr, Thomas M. Record
المصدر: JMB Online (Journal of Molecular Biology); August 2, 1996, Vol. 260 Issue: 5 p697-717, 21p
-
8دورية
المؤلفون: Frank, Diane E., Saecker, Ruth M., Bond, Jeffrey P., Capp, Michael W., Tsodikov, Oleg V., Melcher, Sonya E., Levandoski, Mark M., Record, M. Thomas
المصدر: JMB Online (Journal of Molecular Biology); April 18, 1997, Vol. 267 Issue: 5 p1186-1206, 21p
-
9دورية أكاديمية
المؤلفون: Dworzak, Johannes, Frank, Diane E., Charleston, Jay S., Schnell, Frederick J., Voss, Jon, Donoghue, Cas, Milici, Anthony J., Aeffner, Famke, Young, G. David, Moore, Steven A., Lewis, Sarah, Peterson, Ellyn, Sahenk, Zarife, Shontz, Kimberly, Rodino-Klapac, Louise R., Mendell, Jerry R.
المصدر: European Journal of Paediatric Neurology; Jun2017 Supplement 1, Vol. 21, pe93-e94, 1p
-
10دورية أكاديمية
المؤلفون: Frank DE; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Schnell FJ; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Akana C; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., El-Husayni SH; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Desjardins CA; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Morgan J; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Charleston JS; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Sardone V; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Domingos J; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Dickson G; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Straub V; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Guglieri M; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Mercuri E; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Servais L; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK., Muntoni F; From Sarepta Therapeutics (D.E.F., F.J.S., C.A., S.H.E.-H., C.A.D., J.S.C.), Cambridge, MA; University College London (J.M., V.S., J.D., F.M.); Centre of Gene and Cell Therapy and Centre for Biomedical Sciences (G.D.), Royal Holloway, University of London, Egham, Surrey; Newcastle University John Walton Muscular Dystrophy Research Centre and the Newcastle Hospitals NHS Foundation Trust (V.S., M.G.), Newcastle upon Tyne, UK; Paediatric Neurology and Centro Clinico Nemo (E.M.), Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy; Institute I-Motion (L.S.), Hôpital Armand-Trousseau, Paris, France; Neuromuscular Reference Center (L.S.), CHU Liège, Belgium; Great Ormond Street Hospital (F.M.); and NIHR Great Ormond Street Hospital Biomedical Research Centre (F.M.), London, UK. f.muntoni@ucl.ac.uk.
مؤلفون مشاركون: SKIP-NMD Study Group
المصدر: Neurology [Neurology] 2020 May 26; Vol. 94 (21), pp. e2270-e2282. Date of Electronic Publication: 2020 Mar 05.
نوع المنشور: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Multicenter Study; Randomized Controlled Trial; 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/*biosynthesis , Muscular Dystrophy, Duchenne/*drug therapy , Oligonucleotides/*therapeutic use, Administration, Intravenous ; Adolescent ; Child ; Dose-Response Relationship, Drug ; Double-Blind Method ; Dystrophin/genetics ; Fluorescent Antibody Technique ; Humans ; Male ; Muscle, Skeletal/metabolism ; Muscular Dystrophy, Duchenne/blood ; Muscular Dystrophy, Duchenne/genetics ; Sequence Deletion/drug effects