Francisca Martinez-Raub

This workshop will explore the shared genetic and molecular mechanisms underlying Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD), providing insights into converging pathways that may inform novel therapeutic strategies. Participants will examine the latest discoveries in disease causing mutations, modifier genes, and cryptic exon biology, and discuss how these findings are guiding target identification and translational research. The session will also cover approaches to leverage genetic insights for biomarker development, patient stratification, and the design of mechanism-based clinical trials. Attendees will gain a clearer understanding of the genetic intersections between ALS and FTD and the implications for next-generation therapies.

Key Discussion Points

• Genetic overlap between ALS and FTD and how shared pathways contribute to disease progression
• Insights from discovery programs and translational research into novel targets and modifier genes
• Integration of genetic findings into biomarker development and patient stratification for clinical trials
• Opportunities and challenges in leveraging genetic insights to design mechanism-based therapies across ALS and FTD