Patricia Inácio, PhD, science writer —

Investigational therapy BPN14770 has received orphan drug status from the U.S. Food and Drug Administration as a treatment for fragile X syndrome, Tetra Discovery Partners recently announced. BPN14770, a small molecule therapeutic agent developed by Tetra Discovery, inhibits the phosphodiesterase type-4D (PDE4D) enzyme linked to memory formation and learning…

The gene-editing technology CRISPR/Cas9 was able to restore the expression of the FMR1 gene in nerve cells, the underlying cause of fragile X syndrome. The study “Rescue of Fragile X syndrome neurons by DNA methylation editing of…

Loss of the fragile X mental retardation-1 (FMR1) gene is linked to an early decline in reproduction capacity due to high levels of the protein mTOR, a recent study using mice reveals. These results suggest that mTOR can become a potential therapeutic target. The study “Premature recruitment of oocyte…