Author Archives: Marta Figueiredo PhD

Artificial Intelligence Tool May Help Speed Diagnosis

An artificial intelligence (AI) model based only on the occurrence of health conditions previously and newly associated with fragile X syndrome accurately identified fragile X patients five years earlier than diagnosis, according to a study based on nearly 5,600 people in Wisconsin. Among early signs of fragile X were not…

Zygel Led to Meaningful Behavioral Improvements in Certain Youngsters

Zynerba Pharmaceuticals’ experimental cannabidiol (CBD) gel Zygel results in statistically significant and clinically meaningful reductions in behavioral symptoms in children and adolescents with fragile X syndrome (FXS) and a fully methylated FMR1 gene, according to data from the Phase 2/3 CONNECT-FX trial. DNA methylation is a biochemical modification that…

BPN14770 Improved Men’s Language, Daily Functions

Tetra Therapeutics’ investigational oral therapy BPN14770 safely and effectively improves cognitive function, particularly language-related domains, and daily functioning in men with fragile X syndrome, according to data from a Phase 2 clinical trial. “These results offer hope for patients with fragile X syndrome and their families,” Elizabeth…

Parkinson’s Therapy May Hold Promise for Fragile X

Treatment with istradefylline — an approved therapy for Parkinson’s disease — normalized several molecular and functional nerve cell features of fragile X syndrome and improved cognitive skills in a mouse model of the disease, a study shows. The therapy, sold by Kyowa Hakko Kirin Pharma under the brand name …