2019 FAST Science Summit

Tim tells the story of Mila, a young girl with a rare, fatal, neurodegenerative condition called Batten disease, for whom the Yu Lab developed an experimental ASO therapy personalized to her specific genetic variant.

This presentation recounts the aggressive timeline from ASO development to clinical trial implementation, the challenges present for both researchers and families integrated in the rare disease community, and what these advances might mean for personalized medicine in the future.