In the spirit of Rare Disease Day, we are both reflecting on the past — in particular, the work we started with milasen two and a half years ago — and are looking toward the future…
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Today marks the publication of our manuscript, “Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease,” in the New England Journal of Medicine on October 9, 2019. This article describes an example of truly individualized genomic medicine: the development of a customized oligonucleotide treatment for a young girl with a rare, progressive, neurodegenerative disease
Read MoreTim and Julia Vitarello shared Mila’s story at the GoldLab Symposium.
Read MoreThank you to the American Academy of Neurology for giving us a platform to share our research.
Read MoreTim 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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