News

Zygel, an experimental cannabidiol gel developed by Zynerba Pharmaceuticals to ease abnormal behaviors in people with fragile X syndrome, was well tolerated and effective, including in patients with severe disease, according to a new interim analysis of an open-label extension study. The poster, titled “Long-Term Safety and Sustained Efficacy…

Nova Mentis has received the green light from the U.S. Drug Enforcement Administration (DEA) to ship psilocybin (NM-1001) into Canada, moving it a step closer to being tested as a treatment for fragile X syndrome. Psilocybin arrived recently at the labs of the Toronto Institute of Pharmaceutical…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NeuroNascent’s NNI-351 for the treatment of fragile X syndrome. NNI-351, given orally, is able to penetrate the brain, where it has been shown to boost the proliferation and growth of immature progenitor cells into mature…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Neuronascent‘s investigational therapy NNI-351 for the treatment of fragile X syndrome (FXS). “With vast unmet need for novel therapies that can directly address pediatric FXS behavioral deficits, such as to improve education and social…

In an effort to both increase accuracy and remove offensive language in the name of FMR1 — the gene at the root of fragile X syndrome — the European Fragile X Network (EFXN) led a push to rename it Fragile X Messenger Ribonucleoprotein 1. The name change now…

In people with fragile X-associated tremor/ataxia syndrome (FXTAS), a condition related to fragile X syndrome that develops late in life, measures of dexterity and memory tend to worsen markedly over time, a new study shows. The results support the development of preventive treatments for FXTAS, according to its researchers.

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

Unusually low amounts of cholesterol in cellular structures called lipid rafts may contribute to behavioral differences seen in people with fragile X syndrome, a new study indicates. “The correlation between [lipid rafts] cholesterol and the clinical profile showed a significant association with autistic traits,” the researchers wrote. The study, “…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…