Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double‐knockout mice von Putten, Maaike, Hulsker, Margriet, Young, Courtney, Nadarajah, Vishna D., Heemskerk, Hans, Weerd, Louise, 't Hoen, Peter A. C., Ommen, Gert‐Jan B., Aartsma‐Rus, Annemieke M.

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Cross-sectional study into age-related pathology of mouse models for limb girdle muscular dystrophy types 2D and 2F von Verhaart, Ingrid E C, Putker, Kayleigh, van de Vijver, Davy, Tanganyika-de Winter, Christa L, Pasteuning-Vuhman, Svetlana, Plomp, Jaap J, Aartsma-Rus, Annemieke M, van Putten, Maaike

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Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and caregivers’ perspective von Verhaart, Ingrid E.C., Johnson, Alex, Thakrar, Sejal, Vroom, Elizabeth, De Angelis, Fernanda, Muntoni, Francesco, Aartsma-Rus, Annemieke M., Niks, Erik H.

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Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy von Jirka, Silvana M.G., ’t Hoen, Peter A.C., Diaz Parillas, Valeriano, Tanganyika-de Winter, Christa L., Verheul, Ruurd C., Aguilera, Begona, de Visser, Peter C., Aartsma-Rus, Annemieke M.

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Environmental 24-hr Cycles Are Essential for Health von Lucassen, Eliane A., Coomans, Claudia P., van Putten, Maaike, de Kreij, Suzanne R., van Genugten, Jasper H.L.T., Sutorius, Robbert P.M., de Rooij, Karien E., van der Velde, Martijn, Verhoeve, Sanne L., Smit, Jan W.A., Löwik, Clemens W.G.M., Smits, Hermelijn H., Guigas, Bruno, Aartsma-Rus, Annemieke M., Meijer, Johanna H.

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Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy von Doorenweerd, Nathalie, Mahfouz, Ahmed, van Putten, Maaike, Kaliyaperumal, Rajaram, t’ Hoen, Peter A. C., Hendriksen, Jos G. M., Aartsma-Rus, Annemieke M., Verschuuren, Jan J. G. M., Niks, Erik H., Reinders, Marcel J. T., Kan, Hermien E., Lelieveldt, Boudewijn P. F.

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Author Correction: Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy von Doorenweerd, Nathalie, Mahfouz, Ahmed, van Putten, Maaike, Kaliyaperumal, Rajaram, t’ Hoen, Peter A. C., Hendriksen, Jos G. M., Aartsma-Rus, Annemieke M., Verschuuren, Jan J. G. M., Niks, Erik H., Reinders, Marcel J. T., Kan, Hermien E., Lelieveldt, Boudewijn P. F.

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Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2 von Lemmers, Richard J L F, Tawil, Rabi, Petek, Lisa M, Balog, Judit, Block, Gregory J, Santen, Gijs W E, Amell, Amanda M, van der Vliet, Patrick J, Almomani, Rowida, Straasheijm, Kirsten R, Krom, Yvonne D, Klooster, Rinse, Sun, Yu, den Dunnen, Johan T, Helmer, Quinta, Donlin-Smith, Colleen M, Padberg, George W, van Engelen, Baziel G M, de Greef, Jessica C, Aartsma-Rus, Annemieke M, Frants, Rune R, de Visser, Marianne, Desnuelle, Claude, Sacconi, Sabrina, Filippova, Galina N, Bakker, Bert, Bamshad, Michael J, Tapscott, Stephen J, Miller, Daniel G, van der Maarel, Silvère M

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Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants von van den Bergen, Janneke C, Hiller, Monika, Böhringer, Stefan, Vijfhuizen, Linda, Ginjaar, Hendrika B, Chaouch, Amina, Bushby, Kate, Straub, Volker, Scoto, Mariacristina, Cirak, Sebahattin, Humbertclaude, Véronique, Claustres, Mireille, Scotton, Chiara, Passarelli, Chiara, Lochmüller, Hanns, Muntoni, Francesco, Tuffery-Giraud, Sylvie, Ferlini, Alessandra, Aartsma-Rus, Annemieke M, Verschuuren, Jan J G M, 't Hoen, Peter AC, Spitali, Pietro

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Therapeutic NOTCH3 cysteine correction in CADASIL using exon skipping: in vitro proof of concept von Rutten, Julie W, Dauwerse, Hans G, Peters, Dorien J M, Goldfarb, Andrew, Venselaar, Hanka, Haffner, Christof, van Ommen, Gert-Jan B, Aartsma-Rus, Annemieke M, Lesnik Oberstein, Saskia A J

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The future of exon skipping for Duchenne muscular dystrophy von Aartsma-Rus, Annemieke M

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Longitudinal follow-up of verbal span and processing speed in Duchenne muscular dystrophy von Hellebrekers, Danique M.J., Doorenweerd, Nathalie, Sweere, Dirk J.J., van Kuijk, Sander M.J., Aartsma-Rus, Annemieke M., Klinkenberg, Sylvia, Vles, Johan S.H., Hendriksen, Jos G.M.

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Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain von Zalachoras, Ioannis, Evers, Melvin M, van Roon-Mom, Willeke M C, Aartsma-Rus, Annemieke M, Meijer, Onno C

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A 3-base pair deletion, c.9711_9713del, in DMD results in intellectual disability without muscular dystrophy von de Brouwer, Arjan P M, Nabuurs, Sander B, Verhaart, Ingrid E C, Oudakker, Astrid R, Hordijk, Roel, Yntema, Helger G, Hordijk-Hos, Jannet M, Voesenek, Krysta, de Vries, Bert B A, van Essen, Ton, Chen, Wei, Hu, Hao, Chelly, Jamel, den Dunnen, Johan T, Kalscheuer, Vera M, Aartsma-Rus, Annemieke M, Hamel, Ben C J, van Bokhoven, Hans, Kleefstra, Tjitske

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Delivery of oligonucleotide‐based therapeutics: challenges and opportunities von Hammond, Suzan M, Aartsma‐Rus, Annemieke, Alves, Sandra, Borgos, Sven E, Buijsen, Ronald A M, Collin, Rob W J, Covello, Giuseppina, Denti, Michela A, Desviat, Lourdes R, Echevarría, Lucía, Foged, Camilla, Gaina, Gisela, Garanto, Alejandro, Goyenvalle, Aurelie T, Guzowska, Magdalena, Holodnuka, Irina, Jones, David R, Krause, Sabine, Lehto, Taavi, Montolio, Marisol, Van Roon‐Mom, Willeke, Arechavala‐Gomeza, Virginia

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The NOTCH3 score: a pre-clinical CADASIL biomarker in a novel human genomic NOTCH3 transgenic mouse model with early progressive vascular NOTCH3 accumulation von Rutten, Julie W, Klever, Roselin R, Hegeman, Ingrid M, Poole, Dana S, Dauwerse, Hans G, Broos, Ludo A M, Breukel, Cor, Aartsma-Rus, Annemieke M, Verbeek, J Sjef, van der Weerd, Louise, van Duinen, Sjoerd G, van den Maagdenberg, Arn M J M, Lesnik Oberstein, Saskia A J

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226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherland... von Aartsma-Rus, Annemieke, Ferlini, Alessandra, McNally, Elizabeth M., Spitali, Pietro, Sweeney, H. Lee, Aartsma-Rus, Annemieke M., Szigyarto, Christina Al-Khalili, Bello, Luca, Bronson, Abby, Brown, Kristy, Buccella, Filippo, Chadwick, Jessica, Ferlini, Alessandra, Frank, Diane, Hoffman, Eric, Larkindale, Jane, McClorey, G., McNally, Elizabeth, Munschauer, Rick, Muntoni, Francesco, Owens, Jane, Schara, Ulrike, Spitali, Pietro, Straub, Volker, Sweeney, Lee, Tinsley, Jon, Versnel, Jenny, Vroom, Elizabeth, Welch, Ellen

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226th ENMC International Workshop von Aartsma-Rus, Annemieke, Ferlini, Alessandra, McNally, Elizabeth M., Spitali, Pietro, Sweeney, H. Lee, Aartsma-Rus, Annemieke M., Szigyarto, Christina Al-Khalili, Bello, Luca, Bronson, Abby, Brown, Kristy, Buccella, Filippo, Chadwick, Jessica, Frank, Diane, Hoffman, Eric, Larkindale, Jane, McClorey, G., McNally, Elizabeth, Munschauer, Rick, Muntoni, Francesco, Owens, Jane, Schara, Ulrike, Straub, Volker, Sweeney, Lee, Tinsley, Jon, Versnel, Jenny, Vroom, Elizabeth, Welch, Ellen

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226 th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20-22 January 2017, Heemskerk, The Netherlan... von Aartsma-Rus, Annemieke, Ferlini, Alessandra, McNally, Elizabeth M, Spitali, Pietro, Sweeney, H Lee

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Exon Skipping: Methods and Protocols von Aartsma-Rus, Annemieke

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