Armando Anido, Chief Executive Officer at Zynerba Pharmaceuticals discusses Fragile X syndrome, a genetic condition involving changes in part of the X chromosome. This condition causes a range of developmental problems including learning disabilities and cognitive impairment.
Fragile X syndrome is the most common form of inherited intellectual disability in males and a significant cause of intellectual disability in females. Other signs and symptoms may include symptoms of autism spectrum disorders, seizures, and characteristic physical features. Fragile X syndrome is caused by a change (mutation) in the FMR1 gene and is inherited in an X-linked dominant manner. There is no cure yet and treatment is based on the symptoms present in the person. Early physical and education therapy is recommended.
Approximately 71,000 U.S. patients are believed to have Fragile X syndrome, based on Fragile X syndrome prevalence estimates of approximately 1 in 3,600 to 4,000 in males and of approximately 1 in 4,000 to 6,000 in females.
The Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX) is evaluating the efficacy and safety of CBD transdermal gel (ZYN002) in children ages 3 through 17 years with Fragile X Syndrome. There is currently no therapeutic option approved for the treatment of behavioral symptoms associated with FXS.