In Genetic Disorders, One Size Does Not Fit All.

In drug development, preclinical studies often do not predict human benefit. Why not, then, go right to the source-patients with rare diseases and, more importantly, with specific various genetic mutations that, in aggregate, define the phenotypic clinical disorder? In this issue, Genova et al. describe how induced pluripotent stem cells from patients with two genetic disorders (ataxia-telangiectasia and Aicardi-Goutières syndrome) can be used to better predict responses to immunosuppressive therapy.

A randomized, double-blind, 6-week, dose-ranging study of pregabalin in patients with restless legs syndrome.

Objective: This study evaluated the dose-related efficacy and safety of pregabalin in patients with idiopathic restless legs syndrome (RLS).

Methods: This six-arm, double-blind, placebo-controlled, dose-response study randomized patients (N=137) with moderate-to-severe idiopathic RLS in an equal ratio to placebo or pregabalin 50, 100, 150, 300, or 450 mg/day. The dose-response was characterized using an exponential decay model, which estimates the maximal effect (E(max)) for the primary endpoint, the change in the International Restless Legs Study Group Rating Scale (IRLS) total score from baseline to week 6 of treatment.

Sertraline in children and adolescents with major depressive disorder.

Objective: To explore time to first response and time to first persistent response of sertraline versus placebo and compare these parameters between children (6-11 years old, n = 177) and adolescents (12-17 years old, n = 199) with major depressive disorder.

Method: A 10-week placebo-controlled treatment was followed by a 24-week open-label sertraline treatment. The double-blind studies were not powered to detect efficacy differences between age groups.

Pediatric Quality of Life Enjoyment and Satisfaction Questionnaire (PQ-LES-Q): reliability and validity.

Objective: The pediatric version of the Short Form of the Quality of Life Enjoyment and Satisfaction Questionnaire (PQ-LES-Q) was developed to aid in the assessment of an important aspect of life experience in children and adolescents.

Method: The reliability and validity of the PQ-LES-Q was tested using data from a sample of 376 outpatient children (6-11 years old) and adolescents (12-17 years old) with major depressive disorder.

Results: The internal consistency coefficients at screening, baseline, and endpoint were high (0.