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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

The DNA of these so-called ‘genetic superheroes’ may contain clues about how to treat severe disease. Whether these people were truly naturally resistant to severe disease, and why that might be the case, remains a mystery. Why should research be any different? Giving participants something in return.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.