Arbaclofen drugs and thin X chromosome syndrome

Researchers at MIND Psychiatry Research Institute, University of UC Davis, USA, are participating in a nationwide clinical trial with the participation of many health centers to study about the effect of Arbaclofen. Research shows positive results in the treatment of fragile X chromosomal syndrome in adolescents and adults.

" Arbaclofen is essential in improving the main symptoms of the fragile X chromosome syndrome, helping patients and families achieve a significant improvement in quality of life, " said Randi Hagerman, Director of MIND Psychiatry Research Institute, of UC Davis University, USA, and a leading investigator in the above clinical trial.

Dr. Randi Hagerman (right) and child

This drug is called STX209 , or Arbaclofen . This is a B-type gamma -amino butyric acid receptor agonist (GABA-B) developed by Seaside Therapeutics, Cambridge, Mass., USA.

Researchers will measure the efficacy, safety and tolerability of Arbaclofen in patients with fragile X chromosomal syndrome, aged between 12 and 25, who will Receiving medication or placebo is accompanied by a follow-up period. Researchers at MIND Psychiatry Research Institute, at UC Davis University, USA, will enroll 12 participants.

The fragile X chromosomal syndrome is the leading cause of genetic disease in people with intellectual disabilities and a mutation of a single gene and autism . It is the result of a mutation of a single gene, which slows the development of the fragile X chromosome Syndrome1 (FMR1 gene) on the X chromosome.

The FMR1 gene produces a protein necessary for normal brain development and activity. While patients with fragile X chromosome syndrome lack this protein, as a result, most patients are significantly affected by mental retardation and require lifelong care.

However, currently the US Food and Drug Administration (FDA) has not yet approved the treatment process for patients with fragile X chromosome syndrome with Arbaclofen.