10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases
XTalks
APRIL 24, 2023
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. How Can Study Protocols Be More Effective?
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