Fighting rare diseases: Finding treatments and bringing hope to families
The majority of rare diseases affect children, most of whom have an underlying genetic cause for their condition that is incurable.
Often, their own doctors have never heard of their disease, let alone know how to treat it.
But there is someplace they can turn to for help. The Human Genetics Program at Sanford Burnham Prebys provides insights into the genes and environmental factors that play a role in the development of childhood diseases. Their work often leads to better ways to diagnose, treat, and sometimes, even cure children.
On March 18, 2021, two patients whose lives were saved by discoveries made by Hudson Freeze, Ph.D., and José Luis Millán, Ph.D., joined the scientists for a conversation about what this work means to them and how their lives have been impacted. Watch the full discussion below.