It’s Not Big Data That Matters – it’s Broad Data

Big data will never be an option for rare disease research. By definition, rare diseases affect around 620 people per million.  Ultra-rare diseases may affect as few as 1 in a million. When you have a tiny population, scattered across the globe, how can you hope to find enough clinical trial participants to demonstrate efficacy…

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Rare Shouldn’t Mean Alone

A study published in the Journal of Genetic Counseling provided evidence for something every rare disease patient and caregiver has learned in the school of experience. Alone in a Crowd? Parents of Children with Rare Diseases Experience of Navigating the Healthcare System 1 reported on the challenges rare disease parents face once their children are…

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