July is Fragile X Awareness Month, and patients, their families, advocates, researchers, and healthcare professionals are coming together — in a variety of efforts, including a Pokémon tournament — to increase understanding and spread knowledge of fragile X syndrome, a rare genetic condition that supporters say remains unfamiliar to…
News
Zatolmilast, Shionogi’s experimental oral therapy, was not superior to a placebo in improving key measures of cognition, including verbal knowledge and language skills, in boys and men with fragile X syndrome (FXS) in two late-stage clinical trials. The results come from two U.S.-based Phase 3 studies,…
Suppressing the bone morphogenetic protein (BMP) signaling pathway specifically in astrocytes, the star-shaped brain cells that support neurons, may help reduce seizure severity in fragile X syndrome, a mouse study shows. “Seeing that targeting the BMP pathway in astrocytes alleviated some [fragile X] symptoms makes us optimistic about astrocytes…
Marvel Biosciences plans to launch an early clinical trial in the coming months to test MB-204, its lead therapy candidate for fragile X syndrome and other neurological conditions, in healthy volunteers. The Phase 1 trial will be possible due to funding and expert guidance from 5 Horizons…
The FRAXA Research Foundation will provide financial support to the principal investigators involved in a planned Phase 2b clinical trial of SPG601, an experimental oral therapy being developed for people with fragile X syndrome. “We are thrilled to have the support of FRAXA as part of our journey…
Six months of daily treatment with the diabetes medication metformin was significantly better than a placebo at easing hyperactivity and sleep problems in boys with fragile X syndrome, according to the results of a small clinical trial. However, while those given metformin showed greater overall behavioral improvement than…
Researchers at the University of South Carolina (USC) have received $6.3 million in federal funding to study how fragile X premutations — genetic mutations that do not cause overt symptoms of fragile X syndrome but can still lead to health problems — affect children and adults. The Center of…
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…
Recent Posts
- Advocates aim to answer the question ‘Fragile what?’ this awareness month
- Fragile X drug zatolmilast misses main goal in Phase 3 trials
- Targeting star-shaped brain cells reduces fragile X seizures in mice: Study
- Marvel planning trial to test fragile X treatment in healthy volunteers
- FRAXA backs upcoming Phase 2b trial of SPG601 for fragile X syndrome
- Daily use of diabetes drug metformin eases hyperactivity in fragile X boys
- USC researchers net $6.3M federal grant to study fragile X mutations
- 1st patient enrolled in Phase 2 trial of treatment for fragile X syndrome
- Phase 2 trial of experimental oral treatment enrolling fragile X males
- SPG601 calms brain activity in men with fragile X, helping them to focus