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.

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.

Long-term sertraline treatment of children and adolescents with major depressive disorder.

Objective: The aim of this study was to assess the long-term safety, tolerability, and efficacy of sertraline 50-200 mg once-daily in children (6-11 year olds) and adolescents (12-18 year olds) with a Diagnostic and Statistical Manual of Mental Disorders, 4th edition (DSM-IV) diagnosis of major depressive disorder (MDD).

Methods: This study consisted of a 24-week open-label observational study of children and adolescents who had completed either of two 10-week double-blind, placebo-controlled trials. The Children's Depression Rating Scale-Revised (CDRS-R) was the primary measure of efficacy.