News, Events & Blogs
NIH's All of Us Program Reaches a Historic Milestone for Precision Medicine
The NIH's All of Us Research Program has reached a historic milestone, becoming the world's largest integrated genomics and health database. Learn how this achievement could accelerate rare disease research, improve precision medicine, and bring new hope to patients and families worldwide.
Drug Repurposing: Finding New Uses for Old Medicines in Rare Diseases
For millions living with rare diseases, the biggest challenge isn’t diagnosis — it’s finding effective treatment. Developing a new drug can take over a decade and cost billions, often making it impractical for small patient populations. Drug repurposing offers a faster, lower-risk solution by identifying new uses for existing, safety-tested medications. This approach has already transformed drugs originally developed for other conditions into life-changing therapies for rare genetic, metabolic, and neurodegenerative disorders.
The Future of Rare Disease Research
Rare diseases may affect only a handful of people, but together they impact more than 400 million people worldwide—that’s roughly 1 in 17 people. Historically, they’ve been neglected because of small patient populations and fragmented research efforts. But today, thanks to new technologies and global collaboration, the future of diagnosis and treatment has never looked brighter.