Tim had the opportunity to share Mila’s story with her mother Julia at the Broad Institute Rare Disease Day Symposium — Focus on Rare Disease: Decoding genomes, unlocking therapies.
Read MoreTim 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.
Read MoreTim presented our work on patient-customized oligonucleotide therapy in the Late Breaking Abstract Plenary Session. Cynthia presented a poster (and winner of a Readers’ Choice Award!) on her Rapid Exome NICU Sequencing project.
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