11th Annual Rare Disease Day | The Parents’ Journey Through Drug Development: The Angelman Syndrome Story of Venture Philanthropy

Rare Disease Day is an international event held in late February to raise awareness about the impact on patients and need for research.

On February 25, 2026, the ӳ��ý’s Ladders to Cures Scientific Accelerator in collaboration with The Termeer Institute hosted the 11th Annual Rare Disease Day Event: Advancing Neurodegenerative Disease Research, featuring remarks from Anna Greka, Belinda Termeer, and Catharine Smith, in addition to a lineup of speakers from the rare disease space including Mandana Arbab, Hilary Eaton, Kasper Roet, Juliana Gentile, Dan Leonard, Allyson Berent, and Ajamete Kaykas.

Rare genetic diseases are collectively common: they affect 1 in 10 people in North America. While more than 8,000 genes are known to drive these diseases, fewer than 500 have an available treatment. The mismatch in these numbers underscores the urgent need to disrupt the status quo and develop new approaches to deliver precision cures at scale.

The is committed to uncovering the genetic roots of rare diseases and to using those insights to develop new treatments. With projects spanning multiple disease areas, ӳ��ý scientists are using genetics, functional genomics, computational biology, and chemical biology to better understand and ultimately seek effective treatments for rare diseases.

Building on the bold legacy of Henri Termeer, who pioneered groundbreaking treatments for rare diseases, is a nonprofit organization working to connect the world of healthcare innovators until every patient has a cure.