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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.