EPISODE · Jun 5, 2025 · 49 MIN
How One Patient Organization Drives Drug Development
from RARECast
The Children’s Tumor Foundation has been effective in working with drug developers to advance new therapies for neurofibromatosis, a group of rare, genetic conditions that cause tumors to grow on nerves throughout the body. Part of its success has been its ability to get biopharmaceutical companies to reposition assets once in development for other conditions as potential treatments for neurofibromatosis. We spoke to Annette Bakker, CEO of the Children's Tumor Foundation, about the complexities of neurofibromatosis, the foundation's role in advancing research and drug development, and what other patient organizations can learn from its strategic approach.
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What this episode covers
Annette Bakker, CEO of the Children's Tumor Foundation, discusses the complexities of a group of rare genetic conditions that cause tumors to grow on nerves throughout the body, the foundation's role in advancing research and drug development, and what other patient organizations can learn from its strategic approach.
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How One Patient Organization Drives Drug Development
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