BADANIA

Research on KIF1A / KAND is being conducted on many fronts to better understand the disease mechanisms and accelerate the development of effective therapies.

• • Natural History / KOALA Study - patient observation, disease severity scales, biomarkers.
• • Disease models - iPSC cells and animal models for studying mutations.
• • Genotype-phenotype - analysis of how different mutations affect symptoms.
• • New therapies - ASO, gene editing, drug repositioning, small molecules.
• • Structural biology - studying KIF1A structure and function to identify therapeutic targets.

Thanks to this research, we better understand KAND and are getting closer to developing therapies that can truly improve the lives of patients and their families.