Prof. Dr Annemieke Aartsma-Rus is Professor of Translational Genetics at the Leiden University Medical Center (LUMC, the Netherlands) and an internationally recognised expert in RNA therapeutics and rare disease research. Her work has been instrumental in the development of antisense-mediated exon skipping therapies for Duchenne muscular dystrophy, and she has played a leading role in advancing personalised treatments for rare genetic diseases.

Alongside her research, Annemieke is actively involved in several international initiatives, including the Dutch Center for RNA Therapeutics (DCRT) and the N-of-1 Collaborative, which support the development of innovative therapies for patients with rare and ultra-rare conditions. She has authored more than 280 peer-reviewed publications, holds multiple patents, and has received numerous awards recognising her contributions to translational genetics and rare disease research. She is also widely known for her commitment to education, scientific collaboration, and bridging the gap between patients, researchers, regulators, and industry.