UNCRC Series

6, Feb 2020

Rare disease research may help all

The battlefield of rare diseases is a short paper that looks at how rare disease research can improve things for the whole population.  The paper reviews the work of Goldstein and Brown who did research on familial hypercholesterolaemia, a rare disease that can increase the risk of heart disease.  This research later resulted in statins being developed that are now used regularly to help prevent heart and artery disease and the researchers won a Nobel prize for their work.  This is just one example of how research in rare disease can lead to greater scientific understanding and improve life for the whole population.  Funding for rare disease research can have outcomes that reach far beyond the rare disease community.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3790314/ 

Article 38 https://www.unicef.org.au/Upload/UNICEF/Media/Our%20work/childfriendlycrc.pdf

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