Reflections on Clinical Trials in Fragile X Syndrome
作者： Aia E. Jønch , Sébastien Jacquemont
DOI: 10.1016/B978-0-12-804461-2.00020-2
关键词: Symptomatic treatment 、 Pediatrics 、 Fragile X syndrome 、 Psychiatry 、 Clinical trial 、 Medicine
摘要: Abstract Despite intensive research efforts and an increasing number of clinical trials in fragile X syndrome (FXS), the promising results obtained preclinical studies on targeted treatment FXS have not yet been replicated humans. In this chapter we present overview placebo-controlled trials, open-label symptomatic FXS. Inadequate design and/or underpowered cohorts common challenges previous studies. The largest conducted so far mGluR5-negative modulators GABA receptor agonists, but failed to show any efficacy. Future perspectives for related neurodevelopmental disorders are discussed.
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