Vanda Pinto, PhD, science writer —

Anavex2-73 (blarcamesine), an experimental small molecule being developed by Anavex Life Sciences, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) as a potential treatment of fragile X syndrome. This designation is given to investigational treatments for people with rare diseases, defined…

AKT, an enzyme that is involved in multiple cellular processes, is overactivated in neural stem cells that lack the FMRP protein, as is the case in fragile X, leading to the stimulation of a cellular quality control pathway called NMD, according to a new study. Blocking AKT in these…

Ovid Therapeutics announced it is discontinuing work to further develop or test OV101 (gaboxadol) as a potential treatment of fragile X syndrome. Although OV101 was found to be safe and well tolerated in multiple clinical trials, its overall efficacy in these studies fell short, Ovid Therapeutics said in a…