Patricia Inácio, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has given positive feedback to Actinogen’s pre-investigational new drug (pre-IND) submission regarding Xanamem, its experimental oral therapy for fragile X syndrome. In a written response following a pre-IND meeting with Actinogen, the FDA agreed the data gathered and the proposed trial design…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Infants with fragile X syndrome who are fed with breast milk for a minimum of one year have a decreased prevalence of autism, a retrospective study found. However, breast milk consumption was linked to the earlier development of gastrointestinal problems and allergies among these infants, according to the investigator.

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Nova Mentis is launching a clinical study in the U.S. to assess the diversity of gut microbiome — the community of microbes living in the gut — in people with autism spectrum disorder (ASD), including fragile X syndrome, the company announced. The research is intended to help…

Nova Mentis is expanding its preclinical pipeline to test a psychedelic treatment known as PLZ-1013 in an animal model of fragile X syndrome. The psychedelic compound, psilocybin, is being investigated as a potential behavioral therapy for fragile X. Researchers at the Canada-based company are collaborating with…

A nearly $2 million grant from the National Institute of Mental Health, part of the National Institutes of Health (NIH),  will help researchers understand the mechanisms underlying impaired protein production in fragile X syndrome. The awarded project, “Mechanism of Gp1 mGluR-dependent translation and plasticity,” is led by scientists Nien-Pei…

The U.S. Food and Drug Administration (FDA) and Zynerba Pharmaceuticals will meet later this year to discuss the results of the CONNECT-FX trial and next steps regarding a potential regulatory approval of the experimental cannabidiol (CBD) gel Zygel for fragile X syndrome, according to the company.

Shortage of energy due to leaky mitochondria — the powerhouses of cells — contributes to behavioral abnormalities in fragile X syndrome, a study has found. Closure of the leaky mitochondrial membrane with a form of a Parkinson’s disease medicine boosted energy production and reversed autism-like behaviors…