News

The first patient has been enrolled in a Phase 2 clinical trial testing MRM-3379, Mirum Pharmaceuticals’ experimental oral treatment, in boys and men with fragile X syndrome. Top-line results are expected in 2027. The recently launched study, called BLOOM (NCT07209462), is recruiting up to 60 male…

Mirum Pharmaceuticals has launched a proof-of-concept Phase 2 clinical trial to test its experimental oral treatment MRM-3379 in boys and men with fragile X syndrome. The therapy, acquired by the company last year, is designed to restore the disrupted brain signaling that characterizes fragile X syndrome, which…

Spinogenix’s experimental oral therapy SPG601 reduced abnormal high-frequency brain activity in men with fragile X syndrome and increased their selective attention, allowing them to better focus on one type of information while ignoring others. That’s according to full data from a Phase 2a clinical trial (NCT06413537) completed…

A large clinical trial investigating the effects of Harmony Biosciences’ cannabidiol (CBD) gel ZYN002 on behavioral symptoms in children and young adults with fragile X syndrome has failed to meet its primary goal, the developer announced. Data from the Phase 3 trial, called RECONNECT (NCT04977986), showed no…

Children with Fragile X syndrome (FXS) share some traits with those with autism spectrum disorder (ASD), but exhibit several differences in terms of brain’s traffic patterns, according to an MRI study. Associations between certain brain traffic patterns, which reflect different connections between brain regions that actively work together, and scores…

SPG601, an oral treatment candidate now in clinical testing, has been granted orphan drug status for fragile X syndrome in the European Union. This designation, awarded by the European Medicines Agency (EMA), is meant to incentivize companies developing treatments for rare diseases — those affecting fewer than 5 of every…

Supporters worldwide are gearing up to celebrate Fragile X Awareness Month, an initiative held each July to boost recognition of fragile X syndrome and advocate for funding to improve the lives of people living with the rare disease. The monthlong event is to be celebrated alongside the fifth annual…

KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and…

Long-term, daily treatment with the investigational cannabidiol (CBD) gel ZYN002 — also known as Zygel — led to clinically meaningful reductions in irritability and other behavioral symptoms in people with fragile X syndrome. That’s according to up to three years of data from an ongoing open-label extension…

KER-0193, Kaerus Bioscience’s oral treatment candidate for fragile X syndrome, was safe and well tolerated, and showed predictable pharmacological properties, in healthy volunteers, according to new data from a Phase 1 clinical trial. The therapy was also associated with changes in brain activity in regions known to be…