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Research for Individualized Therapeutics in Rare Genetic Disease
Rochester, Minn.,
Jacksonville, Fla.,
Scottsdale/Phoenix, Ariz.
The purpose of this study is to identify patients with rare genetic disorders that may potentially benefit from an ASO therapeutic based on the characteristics of their genetic disease and the causative genetic alterations and seek the research and development opportunities to have them developed, to have the safety and toxicity studies performed with the ultimate goal of treating the individual in a future N of 1 clinical trial.
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