Annemieke Aartsma-Rus

Scientific Advisor at Sarepta Therapeutics

Annemieke Aartsma-Rus, PhD was elected to the Scientific Advisory Board in November 2019.

She is a Professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center, the Netherlands. She has had a visiting professorship with Newcastle University since 2013.

Prof. Aartsma-Rus has extensive experience in the genetics of Duchenne muscular dystrophy, the dystrophin protein and has been involved in the development of the antisense-mediated exon skipping therapy since her PhD research (2000-2005). She has published over 200 papers in the field of Duchennne, exon skipping therapy development and stakeholder collaboration.

She was elected the most influential scientist in the Duchenne field based on contributions in the past 10 years for 5 consecutive times by Expertscape (2015-now). She has received the Duchenne Award from the Duchenne Parent Project in 2011 for her dedication to the field and the EURORDIS Black Pearl Science Award in 2020 for her key role in therapy development for rare diseases and education of rare disease patient advocates.

Prof. Aartsma-Rus has been a member of the TREAT-NMD executive committee since its initiation and has been Chair of the committee from 2013-2016 and from 2019-2020. She is currently the President of the Oligonucleotide Therapeutics Society (2019-2021) and also Chair of the TREAT-NMD Educational Committee and a core member of the TREAT-NMD Advisory Committee for Therapeutics. She is co-editor in chief of Nucleic Acid Therapeutics and associate editor of Journal of Neuromuscular Diseases.

Annemieke Aartsma-Rus completed her bachelor and master’s degrees in biomedical sciences at Leiden University (1995-2000) and obtained her PhD at Leiden University Medical Center in 2005 on the topic of antisense-mediated exon skipping.


  • Scientific Advisor

    Current role