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Fighting Rare Diseases: Finding Treatments – Bringing Hope
Imagine having a disease so rare it doesn’t even have a name. The majority of rare diseases affect children, most of whom have an underlying genetic cause for their condition that is incurable. Although the number of children living with rare disease is relatively small, the impact on the lives of these patients and their families is profound. 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.

Join us as we meet Hudson Freeze, Ph.D., José Luis Millán, Ph.D., and two of the patients whose lives they impacted for a conversation on how their research is saving the lives of children facing rare disease.

About Sanford Burnham Prebys Medical Research Institute
Sanford Burnham Prebys is a preeminent, independent biomedical research institute dedicated to understanding human biology and disease and advancing scientific discoveries to profoundly impact human health. For more than 40 years, our research has produced breakthroughs in cancer, neuroscience, immunology and children’s diseases, and is anchored by our NCI-designated Cancer Center and advanced drug discovery capabilities. For more information, visit us at SBPdiscovery.org or on Facebook at facebook.com/SBPdiscovery and on Twitter @SBPdiscovery.

Mar 18, 2021 11:00 AM in Pacific Time (US and Canada)

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