Get regular updates to your inbox.
With $1.6 million in pre-seed funding, researchers have launched a new biotech company called Aptadir Therapeutics that will develop RNA-based therapies to reactivate specific genes that are silenced in cancer and genetic diseases such as fragile X syndrome. The therapies will be based on a new class of RNA…
Read more
The nonprofit FRAXA Research Foundation has teamed up with Marvel Biosciences to test MB204, a drug that blocks adenosine A2A receptors, in a preclinical model of fragile X syndrome. As part of the collaboration, FRAXA will run the preclinical studies at its…
Fragile X syndrome is a genetic condition associated with cognitive impairment, learning and behavioral challenges, and several physical features. Learn more about the disease here.
Fragile X is caused by mutations in the fragile X mental retardation 1 (FMR1) gene, located in the X chromosome. Learn more about what causes the disease here.
Symptoms differ in severity and occurrence among patients. Life expectancy for fragile X patients is generally normal. Learn more about the symptoms of the disease here.
Get regular updates to your inbox.
