Local dystrophin restoration with antisense oligonucleotide PRO051 JC Van Deutekom, AA Janson, IB Ginjaar, WS Frankhuizen, ... New England Journal of Medicine 357 (26), 2677-2686, 2007 | 1062 | 2007 |
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 | 909 | 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 | 798 | 2006 |
The TREAT‐NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations CL Bladen, D Salgado, S Monges, ME Foncuberta, K Kekou, K Kosma, ... Human mutation 36 (4), 395-402, 2015 | 720 | 2015 |
Theoretic applicability of antisense‐mediated exon skipping for Duchenne muscular dystrophy mutations A Aartsma‐Rus, I Fokkema, J Verschuuren, I Ginjaar, J Van Deutekom, ... Human mutation 30 (3), 293-299, 2009 | 713 | 2009 |
Duchenne muscular dystrophy D Duan, N Goemans, S Takeda, E Mercuri, A Aartsma-Rus Nature Reviews Disease Primers 7 (1), 13, 2021 | 710 | 2021 |
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 | 656 | 2012 |
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 | 627 | 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 | 411 | 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 | 380 | 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 | 376 | 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 | 366 | 2017 |
Therapeutic developments for Duchenne muscular dystrophy IEC Verhaart, A Aartsma-Rus Nature Reviews Neurology 15 (7), 373-386, 2019 | 341 | 2019 |
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications A Aartsma-Rus, GJB Van Ommen Rna 13 (10), 1609-1624, 2007 | 338 | 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 | 329 | 2002 |
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 (1), 281-308, 2015 | 314 | 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 | 265 | 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 | 252 | 2007 |
Assessing functional performance in the mdx mouse model A Aartsma-Rus, M van Putten JoVE (Journal of Visualized Experiments), e51303, 2014 | 236 | 2014 |