Publications

1. Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic Acid Ther. (2020) Feb 11. doi: 10.1089/nat.2020.0845. [Epub ahead of print] PubMed PMID: 32043902.

2. Verhaart IEC, Johnson A, Thakrar S, Vroom E, De Angelis F, Muntoni F, Aartsma-Rus AM, Niks EH. Muscle biopsies in clinical trials for Duchenne muscular dystrophy - Patients' and caregivers' perspective. Neuromuscul Disord. (2019) Aug;29(8):576-584. doi: 10.1016/j.nmd.2019.06.004. Epub 2019 Jun 12. PubMed PMID: 31378431.

3. Verhaart IEC, Aartsma-Rus A. Therapeutic developments for Duchenne muscular dystrophy. Nat Rev Neurol. (2019) Jul;15(7):373-386. doi: 10.1038/s41582-019-0203-3. Review. PubMed PMID: 31147635.

4. Veltrop M, van Vliet L, Hulsker M, Claassens J, Brouwers C, Breukel C, van der Kaa J, Linssen MM, den Dunnen JT, Verbeek S, Aartsma-Rus A, van Putten M. A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. PLoS One. (2018) Feb 21;13(2):e0193289. doi: 10.1371/journal.pone.0193289. eCollection 2018. PubMed PMID: 29466448.

5.  Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic Acid Ther. (2017) Feb;27(1):1-3. doi: 10.1089/nat.2016.0657. Epub 2016 Dec 8. PubMed PMID: 27929755; PubMed Central PMCID: PMC5312460.

6.  Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A,Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Lancet Neurol. (2016) Jul;15(8):882-90. doi: 10.1016/S1474-4422(16)30035-7. Review. PubMed PMID: 27302365.

7.  Gazzoli I, Pulyakhina I, Verwey NE, Ariyurek Y, Laros JF, 't Hoen PA and Aartsma-Rus A. Non-sequential and multi-step splicing of the dystrophin transcript. RNA Biol. (2015), epub Dec 15.

8.  Van Putten M, Hulsker M, Young C, Nadarajah VD, Heemskerk H, van der Weerd L, ‘t Hoen PAC, van Ommen GJB and Aartsma-Rus A. Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double knockout mice. FASEB (2013); 27: 2484-95. 

9.  Goemans N, Tulinius M, van den Akker JT…..Aartsma-Rus A…and van Deutekom JCT. Systemic administration of PRO051 in Duchenne’s muscular dystrophy. N Engl J Med (2011); 364: 1513-22 

10. Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ, van Deutekom JC. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet. (2003) Apr15;12(8):907-14. PubMed PMID: 12668614.

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