Being a caregiver for someone with fragile X syndrome requires navigating intense emotional, physical, and financial demands, a new review study highlights. Based on these findings, researchers are calling for more proactive efforts to support caregivers of people with fragile X. The study also explores how factors like demographics and…
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Marvel Biosciences’ lead experimental oral therapy MB-204 moved multiple measures of behavior and cognitive function toward normal levels in a mouse model of fragile X syndrome (FXS), with some signs of a potential carry-over effect after treatment was discontinued. “This is now the third ASD [autism spectrum disorder]…
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…
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…
Recent Posts
- Study highlights demands of caregiving in fragile X syndrome
- Experimental therapy improves behavior, cognition in fragile X mice
- 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