News

Gaboxadol in Combo Therapy for Fragile X Worth Pursuing: Healx

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…

Q&A With RARE-X Disease Data Platform Founder, Nicole Boice

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…

Rare Disease Day Events Bring Awareness, Equity to Patients

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…

Study Examines Influences, Effects of Pandemic on Girls

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…

More Flexible ABLE Accounts Among ‘Advocacy Day’ Goals of NFXF

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 Shows Promise in Fragile X Rat Model

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…