10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases
XTalks
APRIL 24, 2023
These are, in my view, the most valid comparisons, particularly in inherited diseases where the genotype is obviously the same in each individual patient, and as a consequence, the difference in phenotype after gene therapy is a true difference,” he adds. As a result, there are several EU regulations and directives to follow.
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