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For people with ultra-rare genetic diseases, a diagnosis can come with a devastating realization that there may be no treatment in development because the patient population is too small to attract traditional biopharma investment. Nome is working to change that by combining patient-led drug development with AI-enabled scientific and operational support. Steven Ringel, founder and CEO of Nome and founder of the Kizuna Foundation, discusses how the company is helping families and patient organizations identify viable genetic-medicine options, build development plans, and find qualified partners who can help advance individualized therapies toward the clinic.
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Amerika Serikat
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