When your child gets sick, you hope there will be a treatment available for them.
For many patients with rare disorders, those treatments don't exist.
This situation is made even more tragic when the science exists to develop a treatment, but the cost and complexities of development have slowed or even stopped progress.
Accelerating Innovation for
Rare Diseases
Emil D. Kakkis
We could be doing more with the science we already have...
The CureTheProcess campaign strives to inspire science-driven public policy that will increase the predictability of the regulatory process for rare disease treatments. Our goal is to give even the rarest diseases access to the accelerated approval pathway and put orphan treatments back on the fast track.
Dr. Kakkis Featured on the "PERSPECTIVES" PODCAST SERIES, presented by Children's Rare Disease Network.
Click Here to Listen to Emil’s Interview.
Congress creates FDA review team for rare and neglected disease