NORD and Rosenau Foundation Launch Rare Disease Research Career Platform — National Organization for Rare Disorders, Inc.

Event summary

NORD and Rosenau Family Research Foundation launched Rare Research Launchpad, a free online learning series for students and early-career scientists in rare disease research.
The program uses cystic fibrosis and Krabbe disease as case studies to explore rare disease biology, translational research, and therapy development.
The initiative includes four expert-led virtual sessions and a downloadable Research Exploration Toolkit with career guidance.
Funding for the program is provided by the Rosenau Family Research Foundation, a NORD member organization.

The big picture

The launch of Rare Research Launchpad addresses the critical need for a skilled workforce in rare disease research, an area where only about 600 of the more than 10,000 known rare diseases currently have available treatments. This initiative aligns with broader industry efforts to advance rare disease research and therapy development, leveraging collaborations between patient advocacy organizations, research institutions, and private foundations. The program's focus on practical career guidance and real-world case studies aims to bridge the gap between academic training and industry needs, potentially accelerating the development of new therapies.

What we're watching

Program Scalability: Whether the Rare Research Launchpad can expand to cover additional rare disease research topics and attract a broader audience of participants.
Career Pipeline Impact: How effectively the program can address the shortage of scientists specializing in rare disease research and therapy development.
Funding Sustainability: The pace at which additional funding sources may be secured to support the ongoing development and expansion of the program.