Forest Ray PhD,  —

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The National Fragile X Foundation (NFXF) will host a virtual patient-focused drug development (PFDD) meeting for fragile X syndrome on March 3. To be held from 10 a.m. to 2 p.m. EST, the meeting provides an opportunity for members of the fragile X community to help make sure their…

The National Fragile X Foundation (NFXF) has launched the International Fragile X Premutation Registry to assist and encourage research into conditions caused by so-called premutations in the gene associated with fragile X syndrome. Created with an advisory committee of fragile X professionals, the registry has now been…

A Phase 1 clinical trial is recruiting adults with fragile X syndrome to study the brain processes underlying altered behavior and cognition, according to the FRAXA Research Foundation. Conducted at Stanford University, the study is seeking to enroll approximately 15 males with fragile X, ages 18…

The U.S. Department of Education awarded $2.1 million to the University of California, Davis, MIND Institute to create a four-year college program for students with intellectual disabilities, including those with fragile X syndrome. The goal of the Supported Education to Elevate Diversity, or SEED, Scholar program is to…

Connecta Therapeutics raised €1.7 million (about $2 million) in investments that will enable it to bring a potential fragile X syndrome (FXS) therapy to Phase 1 clinical trials in late 2021, and to Phase 2a stage by 2023. Fragile X is caused by mutations in the FMR1 gene,…

Almost half of emergency department visits for fragile X syndrome result in hospitalizations, according to a nationwide study in the U.S. The finding underscores the importance of surveillance systems capable of identifying and keeping track of fragile X patients as they move through the healthcare system, scientists said. The…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Better communication among different institutions is needed to assist young patients with fragile X syndrome make the transition from school into adult life, according to a small study. Support programs for youth with fragile X lack continuity and complementarity, the results indicated. The study, “Factors Impacting…