Takeaway
There is not a large enough population at risk for the development of genetic prion disease to accurately power efficacy in randomized clinical trials at current time.
Why this matters ?
Current clinical trials for genetic prion disease randomize symptomatic patients; however, once randomized, patients are already in advanced dementia, and drugs may not show any clinical benefit.
Redesigning the approach to clinical trials for genetic prion disease may be essential to providing access to medications.