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Rare Parents Tackling Rare Diseases

It is estimated that rare diseases affect more than 30 million people in the US alone, and between 300 million and 400 million people worldwide. With so many people suffering from rare diseases, you would think biopharma and pharma companies would be heavily invested in finding cures for such a large number of people. But it’s not that straightforward.

Proof-of-Concept Study Advances Potential New Way to Deliver Gene Therapy

Proof-of-Concept Study Advances Potential New Way to Deliver Gene Therapy
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