Risultati della ricerca - Annemieke Aartsma‐Rus

Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23 di Annemieke Aartsma-Rus

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The use of genetically humanized animal models for personalized medicine approaches di Annemieke Aartsma-Rus, Maaike van Putten, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Prop... di Annemieke Aartsma-Rus, Marc Dooms, Yann Le Cam

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Moving neuromuscular disorders research forward: from novel models to clinical studies di Maaike van Putten, Julija Hmeljak, Annemieke Aartsma-Rus, James J. Dowling

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At the heart of genetic disease: an interview with Elizabeth McNally di Elizabeth McNally, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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To dystrophin and beyond: an interview with Louis Kunkel di Louis M. Kunkel, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) di Raffaella Willmann, Joanne Lee, Cathy Turner, Kanneboyina Nagaraju, Annemieke Aartsma-Rus, Dominic J. Wells, Kathryn R. Wagner, Cristina Csimma, Volker Straub, Miranda D. Grounds, Annamaria De Luca, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice di Flavien Bizot, Remko Goossens, Thomas Tensorer, Sergei Dmitriev, Luis Garcia, Annemieke Aartsma-Rus, Pietro Spitali, Aurélie Goyenvalle

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Case Report: The Genetic Diagnosis of Duchenne Muscular Dystrophy in the Middle East di Fouad Alghamdi, Asmaa Al-Tawari, Hadil Alrohaif, Walaa Alshuaibi, Walaa Alshuaibi, Hicham Mansour, Annemieke Aartsma-Rus, André Mégarbané

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Interactions among ryanodine receptor isotypes contribute to muscle fiber type development and function di Alexis A. Chagovetz, Dana Klatt Shaw, Erin Ritchie, Kazuyuki Hoshijima, David J. Grunwald, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa di Jeroen Bremer, Olivier Bornert, Alexander Nyström, Antoni Gostynski, Marcel F Jonkman, Annemieke Aartsma-Rus, Peter C van den Akker, Anna MG Pasmooij

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Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models di Christa L Tanganyika-de Winter, Hans Heemskerk, Tatyana G Karnaoukh, Maaike van Putten, Sjef J de Kimpe, Judith van Deutekom, Annemieke Aartsma-Rus

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Mouse models for muscular dystrophies: an overview di Maaike van Putten, Erin M. Lloyd, Jessica C. de Greef, Vered Raz, Raffaella Willmann, Miranda D. Grounds, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy di Maaike van Putten, Courtney Young, Sjoerd van den Berg, Amanda Pronk, Margriet Hulsker, Tatyana G Karnaoukh, Rick Vermue, Ko Willems van Dijk, Sjef de Kimpe, Annemieke Aartsma-Rus

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Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy di Mirko Signorelli, Burcu Ayoglu, Camilla Johansson, Hanns Lochmüller, Volker Straub, Francesco Muntoni, Erik Niks, Roula Tsonaka, Anja Persson, Annemieke Aartsma‐Rus, Peter Nilsson, Cristina Al‐Khalili Szigyarto, Pietro Spitali

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Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy di Silvana M G Jirka, Christa L Tanganyika-de Winter, Joke W Boertje-van der Meulen, Maaike van Putten, Monika Hiller, Rick Vermue, Peter C de Visser, Annemieke Aartsma-Rus

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Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor di Dwi U Kemaladewi, Svitlana Pasteuning, Joke W van der Meulen, Sandra H van Heiningen, Gert-Jan van Ommen, Peter ten Dijke, Annemieke Aartsma-Rus, Peter AC 't Hoen, Willem M Hoogaars

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A comparison of the bone and growth phenotype of mdx, mdx:Cmah−/− and mdx:Utrn+/− murine models with the C57BL/10 wild-type mouse di Claire L. Wood, Karla J. Suchacki, Rob van 't Hof, Will P. Cawthorn, Scott Dillon, Volker Straub, Sze Choong Wong, Syed F. Ahmed, Colin Farquharson, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP) di A Seda Yılmaz-Eliş, Annemieke Aartsma-Rus, Peter AC 't Hoen, Huma Safdar, Cor Breukel, Bart JM van Vlijmen, Judith van Deutekom, Sjef de Kimpe, Gert-Jan van Ommen, J Sjef Verbeek

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Integrated lipidomic and transcriptomic analyses identify altered nerve triglycerides in mouse models of prediabetes and type 2 diabetes di Phillipe D. O'Brien, Kai Guo, Stephanie A. Eid, Amy E. Rumora, Lucy M. Hinder, John M. Hayes, Faye E. Mendelson, Junguk Hur, Eva L. Feldman, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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