New Drug Discovered to Treat Intellectual Disabilities by Pooja Shete on December 17, 2020 at 12:58 PM
Fragile X Syndrome is a genetic disorder which cause mild to moderate intellectual disability. It is one of the leading causes of autism. A new drug has been discovered which is the first drug for the treatment for Fragile X Syndrome.
The research was conducted as collaboration between University at Buffalo and Tetra Therapeutics.
The drug BPN14770 showed positive results in phase 2 clinical study and improved cognitive function in adult male patients with Fragile X Syndrome.
According to the Centers for Disease Control and Prevention,
Fragile X Syndrome is the most common cause of inherited intellectual disability and there is no cure for it.
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BUFFALO, N.Y. - A new drug discovered through a research collaboration between the University at Buffalo and Tetra Therapeutics took a major step toward becoming a first-in-class treatment for Fragile X Syndrome, a leading genetic cause of autism.
The drug, BPN14770, achieved positive topline results in a phase 2 clinical study. The innovative treatment improved cognitive function in adult male patients with Fragile X Syndrome.
Fragile X Syndrome - a genetic disorder for which there is no cure - is the most commonly known cause of inherited intellectual disability, according to the Centers for Disease Control and Prevention. We are very excited about the results of this study, said Mark Gurney, PhD, founder and chief executive officer of Tetra Therapeutics. In addition to being safe and well tolerated, treatment with BPN14770 led to significant cognitive improvement, specifically in the language domains, and we also saw a clinically meaningful benefit in overall daily