When there’s no commercial incentive to develop gene therapy – hospitals will try to fill the gap

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Individually, rare diseases are rare, but there are so many rare diseases that over 400 million people are affected worldwide. Together, rare diseases aren’t so rare.

Only 5% of rare diseases have an approved treatment so when we find a treatment that works, we must make sure that patients in need can access those treatments. At the moment, this doesn’t always happen.