Trial Design
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Clinical Trials: Inclusion and Exclusion criteria defined.
Clinical Trials: Limited slots - first come, first served and screened re criteria.
A Canavan mom on learning gene therapy was on the horizon and getting information from the company. “Information is being shared the way it needs to be shared.”
Balancing hope and realistic expectations in clinical trials.
Community input is valued but every trial design is unique
Clinical Trials: Efficacy and End Points defined. End points are indicators on the road to efficacy.