Reply to Oberman et  al.

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