Although we are rare, we dare to do the remarkable! There are approximately 7,000 rare diseases affecting over 30 million people in the United States. Cystinosis affects approximately 2,000 people in the world, and because of CRF’s targeted strategy, our funded research has led to two FDA approvals and several clinical trials.
One of the most fulfilling results of CRF’s work is that the research we fund is now helping other diseases and disorders. For instance, the stem cell and gene therapy work by Dr. Stéphanie Cherqui is being applied to diseases like Friedreich’s Ataxia, Danon disease, corneal diseases, kidney diseases, and systemic diseases similar to cystinosis. We have given hope to our community as well as other rare disease communities. Let’s celebrate our accomplishments on Rare Disease Day!
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