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| Systemic administration of PRO051 in Duchenne's muscular dystrophy NM Goemans, M Tulinius, JT van den Akker, BE Burm, PF Ekhart, ... New England Journal of Medicine 364 (16), 1513-1522, 2011 | 904 | 2011 |
| Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading‐frame rule A Aartsma‐Rus, JCT Van Deutekom, IF Fokkema, GJB Van Ommen, ... Muscle & Nerve: Official Journal of the American Association of …, 2006 | 781 | 2006 |
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| Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2 RJLF Lemmers, R Tawil, LM Petek, J Balog, GJ Block, GWE Santen, ... Nature genetics 44 (12), 1370-1374, 2012 | 644 | 2012 |
| Duchenne muscular dystrophy D Duan, N Goemans, S Takeda, E Mercuri, A Aartsma-Rus Nature Reviews Disease Primers 7 (1), 13, 2021 | 620 | 2021 |
| Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy–a literature review IEC Verhaart, A Robertson, IJ Wilson, A Aartsma-Rus, S Cameron, ... Orphanet journal of rare diseases 12, 1-15, 2017 | 591 | 2017 |
| The importance of genetic diagnosis for Duchenne muscular dystrophy A Aartsma-Rus, IB Ginjaar, K Bushby Journal of medical genetics 53 (3), 145-151, 2016 | 384 | 2016 |
| Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients A Aartsma-Rus, AAM Janson, WE Kaman, M Bremmer-Bout, ... Human Molecular Genetics 12 (8), 907-914, 2003 | 373 | 2003 |
| Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense A Aartsma-Rus, AAM Janson, WE Kaman, M Bremmer-Bout, ... The American Journal of Human Genetics 74 (1), 83-92, 2004 | 370 | 2004 |
| FDA approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga A Aartsma-Rus, AM Krieg Nucleic acid therapeutics 27 (1), 1-3, 2017 | 356 | 2017 |
| Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications A Aartsma-Rus, GJB Van Ommen Rna 13 (10), 1609-1624, 2007 | 334 | 2007 |
| Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy A Aartsma-Rus, M Bremmer-Bout, AAM Janson, JT den Dunnen, ... Neuromuscular Disorders 12, S71-S77, 2002 | 330 | 2002 |
| Therapeutic developments for Duchenne muscular dystrophy IEC Verhaart, A Aartsma-Rus Nature Reviews Neurology 15 (7), 373-386, 2019 | 328 | 2019 |
| The pathogenesis and therapy of muscular dystrophies S Guiraud, A Aartsma-Rus, NM Vieira, KE Davies, GJB van Ommen, ... Annual review of genomics and human genetics 16, 281-308, 2015 | 306 | 2015 |
| In vivo comparison of 2′‐O‐methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping HA Heemskerk, CL de Winter, SJ de Kimpe, P van Kuik‐Romeijn, ... The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2009 | 277 | 2009 |
| Current status of pharmaceutical and genetic therapeutic approaches to treat DMD C Pichavant, A Aartsma-Rus, PR Clemens, KE Davies, G Dickson, ... Molecular Therapy 19 (5), 830-840, 2011 | 257 | 2011 |
| Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle V Arechavala-Gomeza, IR Graham, LJ Popplewell, AM Adams, ... Human gene therapy 18 (9), 798-810, 2007 | 250 | 2007 |
| Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms A Aartsma-Rus, L Van Vliet, M Hirschi, AAM Janson, H Heemskerk, ... Molecular Therapy 17 (3), 548-553, 2009 | 235 | 2009 |
