The challenges of clinical trials in fragile X syndrome

• Published in: Psychopharmacology Vol. 231; no. 6; pp. 1237 - 1250
• Main Authors: Jacquemont, Sébastien, Berry-Kravis, Elizabeth, Hagerman, Randi, von Raison, Florian, Gasparini, Fabrizio, Apostol, George, Ufer, Mike, Des Portes, Vincent, Gomez-Mancilla, Baltazar
• Format: Journal Article
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.03.2014; Springer; Springer Nature B.V; Springer Verlag
• Subjects: Analysis; Animals; Autism; Biomedical and Life Sciences; Biomedicine; Care and treatment; Child Development Disorders, Pervasive - drug therapy; Child Development Disorders, Pervasive - physiopathology; Clinical trials; Clinical Trials as Topic - methods; Cognitive science; Developmental disabilities; Diagnosis; Fragile X syndrome; Fragile X Syndrome - diagnosis; Fragile X Syndrome - drug therapy; Fragile X Syndrome - genetics; Fragile X Syndrome - physiopathology; GABA; Genetic aspects; Genetic disorders; Humans; Methylation; Neurosciences; Pharmacology/Toxicology; Phenotype; Psychiatry; Psychopharmacology; Review; FMRP; Disease modification; Mavoglurant; GABA; Fragile X syndrome; mGluR5; Arbaclofen; Autism spectrum disorder; AFQ056

Rationale Advances in understanding the underlying mechanisms of conditions such as fragile X syndrome (FXS) and autism spectrum disorders have revealed heterogeneous populations. Recent trials of novel FXS therapies have highlighted several challenges including subpopulations with possibly differen...