Scientific Information
Topics
Our Scientific Programme brings together leading experts from across the global Pompe disease community to explore the current landscape of Pompe disease, advances in diagnosis and treatment, emerging therapeutic approaches, and key priorities for the future. These topics will be further explored through our scientific sessions and Poster presentations.
Disease landscape, diagnosis & natural history
Infantile- and Late-Onset Pompe Disease
Disease monitoring, imaging & outcome measures
Current and next-generation ERT
Gene therapy & emerging therapeutic approaches
Clinical trials & treatment pipeline
Registries, data & global core dataset
Patient outcomes & shared decision-making
Consensus guidelines & treatment algorithms
Global collaboration & future roadmap
Scientific Coordinator
Professor Dr. Benedikt Schoser
Friedrich Baur Institute, LMU Medizin LMU Munich
Topics
Explore the key topics that will shape the scientific programme of the 1st Meeting of the Global POMPE Consortium.