Clinical SafetyEarly clinical data show proof of concept with no signs of toxic MeCP2 overexpression, bolstering confidence in the gene therapy's safety profile.
Natural History EvidenceComprehensive natural history analyses indicate limited spontaneous milestone gains in the targeted patient group, supporting the planned statistical design's ability to detect a true treatment effect.
Regulatory ValidationRegulatory reviewers endorsed the trial's statistical approach and judged powering calculations to be solid, supporting the study's credibility and regulatory acceptability.