Neurotrope BioScience, Newark, NJ received Orphan Drug Designation from the FDA for bryostatin for the treatment of Fragile X Syndrome (FXS). FXS is a rare genetic disorder that causes a range of developmental problems including learning disabilities and cognitive impairment. The gene whose mutations are responsible for FXS is FMR1. FMR1 gene encodes a protein that is involved in the development of synapses, which are specialized connections between nerve cells. Bryostatin is an activator of Protein kinase C, a protein kinase involves in multiple biochemical pathways. Neurotrope believes that PKC activator is a viable therapeutic approach for the treatment of FXS based on pre-clinical studies. Currently, there is no FDA approved treatment for FXS.