Fragile X Syndrome: Orphan designation for molecule

14 noviembre 2014

The European Medicines Agency (EMA) has granted «orphan designation» to BMS 204352(1), a molecule developed by French researchers to treat Fragile X Syndrome, a rare genetic disease for which there exists no treatment. Fragile X Syndrome is a genetic disease that causes inherited intellectual disability often associated with autism spectrum disorders as well as with characteristic physical signs. Affecting almost one in 4,000 infants, this is one of the most frequent rare diseases, but there is no existing treatment.