Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center (LUMC, the Netherlands). She played an important role in the development of antisense mediated exon skipping for Duchenne muscular dystrophy starting with her PhD research. 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. She is currently chair of the scientific advisory board 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 280 peer-reviewed papers, 12 book chapters and 15 patents. She has received multiple awards, most recently, the Norman Saunders Jacob’s Ladder International Research Prize from the Canadian foundation for the control of neurodegenerative diseases and SickKids Toronto in 2024 and the Ammodo Science Award for her contribution to developing exon skipping therapies for Duchenne.

She is Chair of the TREAT-NMD educational committee (TEC), and member of the Board of Directors of the Oligonucleotide Therapeutics Society (President 2019-2021). She was chair of the TREAT-NMD Advisory Committee for Therapeutics (TACT, 2021-2025) and Chair of the advisory board member of the N-of-1 Collaborative.

She is co-editor in chief of Nucleic Acid Therapeutics, and serves or served on multiple editorial boards, e.g. Journal of Neuromuscular Diseases (associate editor until 2025), Molecular Therapy and Therapeutic Advances in Rare Disease.