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

Connect to this object online.

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

Connect to this object online.

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... by Annemieke Aartsma-Rus, Marc Dooms, Yann Le Cam

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) by 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

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

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

Connect to this object online.

Mouse models for muscular dystrophies: an overview by 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

Connect to this object online.

Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy by 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

Connect to this object online.

Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy by 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

Connect to this object online.

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy by 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

Connect to this object online.

Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor by 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

Connect to this object online.

A comparison of the bone and growth phenotype of mdx, mdx:Cmah−/− and mdx:Utrn+/− murine models with the C57BL/10 wild-type mouse by 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

Connect to this object online.

Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP) by 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

Connect to this object online.

Integrated lipidomic and transcriptomic analyses identify altered nerve triglycerides in mouse models of prediabetes and type 2 diabetes by 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

Connect to this object online.