Free, online learning series and career exploration toolkit introduce aspiring scientists to rare disease research through real-world case studies

NORWELL, Mass., Sept. 15, 2026 /PRNewswire/ — The National Organization for Rare Disorders (NORD®) and the Rosenau Family Research Foundation today launched Rare Research Launchpad: A Career Exploration Series in Rare Disease Research, a free, self-paced online series designed to introduce students and early-career scientists to rare disease research and career opportunities available in the field.

Using cystic fibrosis and Krabbe disease as real-world case studies, Rare Research Launchpad introduces participants to the science behind rare disease research, how discoveries can progress from the laboratory toward potential therapies, and the skills, experiences, and career pathways involved in advancing rare disease research. The free series is available now to students and early-career trainees through NORD’s RareEDU® online learning platform at learn.rarediseases.org.

Funded by the Rosenau Family Research Foundation, a NORD Member Organization, and developed by NORD in collaboration with researchers from the NORD® Rare Disease Centers of Excellence Network, the series combines scientific learning with perspectives from researchers and families affected by rare disease, along with practical career guidance. The series is designed to reach students interested in science and medicine who may have limited exposure to rare disease research or awareness of the career opportunities available within the field. The series can also serve as a supplemental resource for graduate students and early-career trainees pursuing careers in science and medicine.

The Rare Research Launchpad includes:

• Four expert-led virtual sessions featuring clinician-scientists, researchers, and impacted individuals and families
• Case studies in cystic fibrosis and Krabbe disease exploring rare disease biology, translational research, and therapy development
• A downloadable Research Exploration Toolkit with guidance on coursework, skill development, mentorship, internships, conferences, and funding opportunities

“As technologies used to study rare and genetic diseases and develop new therapies continue to advance, we need to prepare the next generation of scientists who will carry this work forward,” said Rebecca Aune, MPA, Director of Education Programs at NORD. “Rare Research Launchpad gives students an early look at rare disease research and helps them understand the skills, experiences, and relationships that can lead to a career advancing science and improving the lives of millions of people with rare diseases.”

Cystic fibrosis and Krabbe disease were selected as case studies because they offer distinct examples of the scientific challenges and opportunities involved in researching rare and genetic diseases. Together, they expose participants to broader principles in genetics, molecular biology, neuroscience, and biomedical research while demonstrating how laboratory science, clinical expertise, and the experiences of patients and families can collectively inform research and the development of new therapies. The two conditions also reflect different points along the therapeutic development landscape: cystic fibrosis has benefited from decades of research that have led to multiple FDA-approved therapies, including treatments that target the underlying disease process, but there is currently no FDA-approved therapy specifically indicated to treat Krabbe disease. Together, these examples highlight both the progress that rare disease research can achieve and the scientific and therapeutic challenges that remain across the broader rare disease field.

“The Rosenau Family Research Foundation is focused on advancing research that can improve the lives of people affected by Krabbe disease and cystic fibrosis,” said Gabriel M. Cohn, MD, MBA, Executive Director and Medical Director of the Foundation. “Supporting NORD’s Rare Research Launchpad allows us to invest in future scientists while giving them a practical understanding of the research questions, career paths, and collaborations needed to advance treatments for the more than 10,000 known rare diseases of which only about 600 currently have an available treatment.”

Rare Research Launchpad is designed as a scalable model that can expand over time to introduce participants to additional rare disease research topics and provide further opportunities for career exploration and engagement.

Rare Research Launchpad is available now at learn.rarediseases.org. Participation is free.

About the National Organization for Rare Disorders

Founded in 1983, the National Organization for Rare Disorders (NORD®) is a leading independent, nonpartisan nonprofit organization dedicated to improving the health and lives of more than 30 million Americans with rare diseases. In partnership with more than 350 disease-specific member patient organizations and 49 designated NORD Rare Disease Centers of Excellence spanning over 170 academic medical centers, research institutions, and children’s hospitals nationwide, NORD advances care, research, education, and advocacy to create a better future for people and families with rare diseases. Learn more at rarediseases.org.

About the Rosenau Family Research Foundation
The Rosenau Family Research Foundation is a 501(c)(3) private family foundation established in 2008 to improve the lives of people impacted by Krabbe disease and cystic fibrosis through research funding and advocacy. The Foundation supports basic, translational, and clinical research, as well as education and awareness efforts that advance treatments, cures, screening, and understanding of both diseases. Learn more at rosenaufoundation.org.

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SOURCE National Organization for Rare Disorders (NORD®)