Healx has entered into an exclusive licensing agreement with Ovid Therapeutics to develop and commercialize gaboxadol in a potential combination treatment for fragile X syndrome and other disorders. The announcement follows Ovid’s decision to discontinue the development of gaboxadol (previously called OV101), despite it being deemed safe and well-tolerated in…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
A survey of women with fragile X syndrome revealed that most identified with a label other than that of the chromosomal disorder, and that the majority either did not want to have a child with the genetic disease or were conflicted. Despite most respondents reporting some learning disabilities, the…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
Differences in pre-pandemic mother-daughter relationships, and social and adaptive behavior significantly predicted worries and impact of the COVID-19 pandemic in school-aged girls with fragile X syndrome (FXS), a U.S. study reports. Notably, this association pattern was distinct from that of matched girls without the condition, whose responses were determined…
Nova Mentis, a Canadian biotechnology company, is teaming up with KGK Science to develop Nova’s portfolio of therapies based on the psychedelic compound psilocybin. The companies are planning to ask Health Canada for the go-ahead to launch a Phase 2 clinical trial in fragile X syndrome that would test…
The ABLE Employment Flexibility Act and better workplace opportunities for people with disabilities are among priorities for this year’s National Fragile X Foundation (NFXF) Advocacy Day, set for March 1. For more than 20 years, the nonprofit organization has brought advocates together in Washington, D.C., to meet…
Gene therapy rescued several behavioral and brainwave abnormalities seen in a rat model of fragile X syndrome. Findings from the study support further development of gene therapy for treating people people with the condition, the scientists said. The study, “Gene Therapy Using an Orthologue of Human…
Nova Mentis and Mycrodose Therapeutics have teamed up to test whether psychedelic treatments, such as Nova’s psilocybin — a potential behavioral therapy for fragile X syndrome — can be delivered via the skin using Mycrodose’s transdermal technology. The first studies assessing the diffusion of psychedelic compounds through the…
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