How Rare Diseases Are Showing the Way Toward Precision Medicine – Part 3

April 18, 2018

In our previous post, we discussed the approaches to help you identify, enroll and retain rare disease populations in clinical trials. Once the right patients have been enrolled in the trial, the challenge becomes measuring the efficacy of the experimental treatment and determining whether it’s producing the desired effect. However, invasive drug delivery methods, such as IV injection, can complicate this measurement and establishing whether the therapy actually reaches the target tissue can be difficult.

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