Patricia Inácio, PhD, science writer —

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…

Measuring the levels of three blood biomarkers could help identify patients with fragile X syndrome who are likely to benefit from treatment with the diabetes medicine metformin, a study suggests. The study, “Blood-Based Biomarkers Predictive of Metformin Target Engagement in Fragile X Syndrome,” was published in the journal…

A parent language intervention based on story-telling eases the severity of speech and social impairments in children with fragile X syndrome, a study shows. The study also found that adding lovastatin, a medication widely prescribed for lowering cholesterol, leads to no additional benefits. The study “Controlled…