Margarida Maia, PhD, science writer —

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…

July is Fragile X Awareness Month, and this year, there are many ways to help build and spread knowledge about fragile X syndrome, collect funds for research, and advocate for those living with the rare genetic disease. To encourage people to stay engaged in the countdown to World Fragile…

Researchers have developed a highly-sensitive test that can reliably detect trace activity levels of FMR1 — the faulty gene in fragile X syndrome (FXS) — in the blood of patients who’d been thought to have none by current methods. This was done through the measurement of FMR1’s messenger RNA…

University of Rochester researcher Lynne E. Maquat, PhD, is co-recipient of this year’s Gruber Genetics Prize for her discovery of nonsense-mediated mRNA decay (NMD), a protein quality control system within cells. Maquat and her team subsequently discovered that this control system is in overdrive in cells of people with…

Girls and women with fragile X syndrome show different learning impairments relative to boys and men with the disease, a finding that was paralleled in a mouse model of the disease, a study found. A feature common across both sexes and species was a difficulty in cognitive flexibility, or…

Capsules containing very small doses (microdoses) of psilocybin are ready to be used in a future Nova Mentis clinical trial, with the company one step closer to testing them as a potential treatment for fragile X syndrome. The capsules started being produced per chemistry, manufacturing, and control (CMC)…

Arbaclofen, an investigational medicine for fragile X syndrome, was found to improve certain aspects of behavior in about half of children, ages 5 to 11, who entered a placebo-controlled Phase 3 clinical trial. The findings unfolded from a new look into trial data by therapy developer Seaside Therapeutics, before…

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…

Wearable motion sensors someday may help doctors diagnose fragile X syndrome and similar disorders based on the way people walk, a study suggests. The method may be able to identify problems in walking 15 to 20 years ahead of clinical symptoms; as such, it offers an opportunity to intervene sooner to…

Nova Mentis is seeking orphan drug designation from the European Medicines Agency (EMA) for psilocybin, its investigational therapy for fragile X syndrome. Psilocybin, found naturally in certain mushrooms — popularly called “magic mushrooms” — is most known for its hallucinogenic, anxiolytic, and psychoactive effects. However, Nova officials…