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 2,500 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 of Dr. Stéphanie Cherqui is being applied to diseases such as Friedreich’s ataxia, Danon disease, corneal and kidney diseases, and systemic diseases such as cystinosis.
We have given hope to our community and to other rare disease communities. Let’s celebrate our accomplishments on Rare Disease Day!
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