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Webinar – Advocacy in rare disease: Driving Technology Advances

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Discover our webinars broadcasted by Science Magazine

The sixth webinar of the 2023 series aired December 7th and is available on demand. 

Register and watch 

About the webinar

By many measures, this is a golden age of drug discovery, particularly for rare diseases. In 2022, more than half of the novel drugs approved by the U. S. Food and Drug Administration were for patients with rare disease. Many of these treatments represent modalities at the cutting edge of medicine. With genomics advances offering pathways to personalized medicine and artificial intelligence transforming pharmaceutical research, people with rare diseases have reason to hope. Yet, in too many cases, conditions remain without diagnostic or treatment options, so advocates continue to make a case for technology advancements that could bring about new therapies. At the same time, ensuring patients have equitable access to the latest advances across the globe remains a challenge as well.

In this Science Webinar, participants will:

  • Explore the pivotal role technology play in advancing diagnostic and treatment options for rare diseases,
  • Hear about the latest breakthroughs in genomics, cell and gene therapies, and digital innovations in rare disease,
  • Learn how advocates are ensuring advances in rare disease technology reach people who need them.

This webinar will last for approximately 60 minutes.



Léon van Wouwe (Volv Global, Epalinges, Switzerland)
Heidi L. Rehm, Ph.D., FACMG (Massachusetts General Hospital, Boston, MA)
Karin Hoelzer, D.V.M., Ph.D. (National Organization for Rare Disorders, Washington, DC)
Simon Alfano (McKinsey & Company, Boston, MA)
Erika Gebel Berg, Ph.D. (Science/AAAS, Washington, DC)
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