Neurotrope Receives FDA Orphan Drug Designation for Bryostatin in the Treatment of Fragile X Syndrome

Industry News

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.


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