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. This is important since not all pharmacies and sites are eligible to prepare and administer gene therapy due to biosafety and technical reasons. There needs to be a mock run done with sites and pharmacies.
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