Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center (the Netherlands). In 2020 she co-founded the Dutch Center for RNA Therapeutics (DCRT), a non-for-profit academic collaboration aiming to develop clinical treatment with exon skipping therapies for eligible patients with unique mutations. In 2022 she became a board member of the N-of-1 collaborative (N1C), a global umbrella organization aiming to facilitate development of antisense oligonucleotide therapies for patients with very rare, eligible mutations.
She has published over 220 peer-reviewed papers, 11 book chapters and 15 patents. She has given many invited lectures at scientific conferences and patient organization meetings. In 2011 she received the Duchenne Award from the Dutch Duchenne Parent Project in recognition of her dedication to the Duchenne field. In 2020 she received the Black Pearl Science Award from Eurordis. In 2021 she received the Ammodo Science Award for her contribution to developing exon skipping therapies for Duchenne, the outstanding achievement award from the Dutch Society of Gene and Cell Therapy the Rosalind Franklin in Science award for her work for the journal Nucleic Acid Therapeutics. Since 2015 she is the most influential scientist in Duchenne muscular dystrophy in the past 10 years (https://expertscape.com/ex/muscular+dystrophy%2C+duchenne).
She is chair of the TREAT-NMD Advisory Committee for Therapeutics (TACT), Chair of the Dutch Center for RNA Therapeutics, vice-chair of COST Action CA17103 (Delivery of antisense RNA therapies), was President of the Oligonucleotide Therapeutics Society (2019-2021), and Chair of the TREAT-NMD executive committee (2013-2016 and 2019-2020). She is a member of the Therapies Scientific Committee of the International Rare Disease Research Consortium (IRDiRC). She is co-editor in chief of Nucleic Acid Therapeutics, and served on the editorial board of Journal of Neuromuscular Diseases, Molecular Therapy and Therapeutic Advances in Rare Disease.