Genotype and Pharmacy - Clinical Research Informer

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

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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. There needs to be a mock run done with sites and pharmacies.

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

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