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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.
Navigating Clinical Trials: A Guide for Rare Disease Patients
A condition is defined as a rare disease if it affects fewer than 200,000 individuals. Approximately 7,000 conditions meet this definition. In 2016, the United States Food and Drug Administration awarded $23 million dollars towards a four year period that supported research for 21 different rare diseases. However, most rare diseases lack disease-modifying therapies or effective medications for several reasons…