Fondation Ipsen

Webinar – Personalized therapies in rare disease

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Discover our webinars broadcasted by Science Magazine

This new free webinar, in partnership with AAAS/Science magazine, will be broadcast on July 7th (noon ET / 6pm CET) and will be available on demand from July 9th.


Watch the webinar

Or listen it as a podcast:

Even though the total number of patients with rare diseases exceeds 300 million, each rare disease affects only a small number of patients. Consequently, the standard model for developing new medicines is often not profitable enough for pharmaceutical companies to produce therapeutics for the thousands of rare diseases known to exist. In the past, pharmaceuticals designed for common diseases have been repurposed for patients with rare diseases. However, that is all changing: New drug design methodologies allow us to contemplate designer medicines for very small groups of patients or even individuals. The future holds promise for the rare disease community because modern biotech can build bespoke (customized) therapies. Are we at the dawn of personalized treatments being widely available for individual patients? This poses a broader question: Just as every person is unique, could all medicines become so too?


Genine Winslow, M.Sc. (Chameleon Biosciences, San Anselmo, CA)
Tiina Urv, Ph.D. (National Institutes of Health, Bethesda, MD)
Viviana Giannuzzi, Pharm.D., Ph.D. (Fondazione Gianni Benzi Onlus, Bari, Italy)
Sean Sanders, Ph.D. (Science/AAAS, Washington, DC)

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