Reply to Oberman et al

We are pleased this article has generated interest and happy to respond to the comments of Oberman et al. They raised both theoretical and specific issues; we will address theory first.1 We agree that an outcome measure for clinical trials must be valid and reliable, however disagree on what that means. Rett syndrome results in both functional impairments and comorbidities that detract from quality of life. Any clinically meaningful improvement must address these in a measurable way, and any clinical trial endpoint must be accurate and reproducible.
Source: Pediatric Neurology - Category: Neurology Authors: Tags: Letter from the Editor Source Type: research