RSBQRett Syndrome Behaviour Questionnaire
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RTT clinical severity was assessed using either the total clinical severity score (CSS), a validated clinical rating specifically designed for RTT, or the "compressed" CSS both based on 13 individual ordinal categories measuring clinical features common in RTT [24], and phenotypical severity was also measured by the use of the Rett Syndrome Behaviour Questionnaire (RSBQ) [25].
Cass, "The Rett Syndrome Behaviour Questionnaire (RSBQ): refining the behavioural phenotype of Rett syndrome," Journal of Child Psychology and Psychiatry and Allied Disciplines, vol.
Neuren conducted a Phase 2 double-blind placebo-controlled dose ranging study in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated on the RSBQ and the CGI-I.