CHALLENGE #1: Development of a non-invasive tool for measuring rare disease patient mobility in daily living
– Digital tools 4 Rare Diseases (DT4RD)
CHALLENGE #2: Delivery system for intranasal administration of biological drugs to neonates
– Intranasal Device for Neonates (INDENEO)
Challenge #4: Pre-clinical assay to detect instability of microsatellite repeat
– Development and validation of a novel pre-clinical assay to detect triplet repeat expansions (TRXassay)