What is the ultimate example of a win in rare disease? A cure.
While a lot of attention has been focused on emerging cell and gene therapies, there are other promising roads to a cure as well, such as repurposed drugs. Many obstacles still exist as the science evolves and access issues remain, but there are a few shining examples of what these new technologies could mean for people living with rare disease.
In this webinar, participants will
Learn about the latest research in curative treatments for rare disease
Hear about first-hand experiences with cures for rare disease
Explore what the future holds for these treatment modalities and what they mean for people with rare disease
Speakers:
Bill Hobbs, PhD, Vertex Pharmaceuticals
Michelle Werner, Alltrna
David Fajgenbaum, PhD, University of Pennsylvania