Trial Design
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Clinical Trials: Inclusion and Exclusion criteria defined.
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Clinical Trials: Limited slots - first come, first served and screened re criteria.
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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.”
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Balancing hope and realistic expectations in clinical trials.
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Community input is valued but every trial design is unique
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Clinical Trials: Efficacy and End Points defined. End points are indicators on the road to efficacy.
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