Education, empowerment aim of International Fragile X Conference

Gathering will be held July 25-28 at the Renaissance Sea World in Florida

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Patients, caregivers, and professionals will gather next month at the National Fragile X Foundation‘s (NFXF) 19th International Fragile X Conference to learn about the latest updates in fragile X syndrome care and research and help empower those living with the rare disease.

The conference will be held July 25-28 at the Renaissance Sea World in Orlando, Florida, and a virtual participation option is available. Those interested in participating have several registration options, with regular pricing available through June 14.

Discounted registration prices are available for self-advocates, fragile X patients at least age 13 who plan to attend self-advocate sessions, and for their siblings, ages 13-17, through a subsidy from NFXF’s Sibling & Self-Advocate Network.

The conference will include presentations, panels, and activities that cover all aspects of fragile X research, care, and lived experiences. There will be two keynote sessions:

  • “Embracing the Xtraordinary: Inspiration from the Fragile X Community” on Friday, July 26, 8:30-9:30 a.m. EST. In this session, Fragile X community members will share how the disease has affected their lives and how they’ve navigated those challenges.
  • “From Past to Future: Charting the Course of Treatments for Fragile X-associated Conditions” on Saturday, July 27, 8:30-9:30 a.m. EST. Here, Peter Todd, MD, the director of University of Michigan’s clinical neurogenetics research program, will discuss the fragile X treatment landscape, its evolution over time, and promising treatments on the horizon.

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Self-advocate sessions, panel of experts

Self-advocate sessions will cover topics and activities aimed at helping fragile X patients feel empowered and facilitate connections with other self-advocates and their caregivers. Some sessions will be interactive, including hands-on activities, music, and talking.

On July 26, a self-advocate session called “Stay Calm and Regulate” will seek to help patients understand how to identify dysregulated emotions and how to manage them. It will include a take-home toolkit.

Later that day, a panel of experts will discuss strategies and skills for success in the workplace in a session called “Panel Discussion-Employment Strategies and Successes.” The next day, an interactive self-advocate session called “Let’s Get Flexible!” will help patients learn about behavioral flexibility and strategies to avoid getting stuck in a pattern.

Other oral and poster sessions will cover updates in fragile X research, clinical trials, and aspects of managing life with fragile X as a patient, caregiver, or sibling. Participants can see the full conference agenda online and filter it to find sessions that interest them.

Virtual participants will be able to livestream most sessions or access recordings afterwards. Self-advocate sessions will not be livestreamed or recorded for privacy reasons.

Events throughout the conference will also enable in-person participants to meet up and network, including a welcome reception on the first day, informal lunches, and a celebration on Saturday night.

SPG601 wins orphan drug status

In other news, early this month the U.S. Food and Drug Administration (FDA) granted orphan drug status to Spinogenix’s experimental oral therapy SPG601 for fragile X. The designation offers financial and regulatory incentives to speed up the development of therapies that address rare diseases, defined in the U.S. as ones affecting fewer than 200,000 people.

“Receiving [orphan drug designation] for SPG601 underscores the high unmet need for new treatment options for individuals with FXS [fragile X syndrome]” Craig Erickson, MD, Spinogenix’s chief medical officer, said in a company press release.

SPG601 is a small molecule designed to bind to and activate large conductance, calcium-activated potassium channels, or BK channels, whose dysfunction has been implicated in driving the symptoms of fragile X.

“This approach aims to address core symptoms of FXS with the goal of significantly improving the lives of those impacted by the condition,” Erickson said.

In mid-April, the FDA cleared Spinogenix to launch a Phase 2 clinical trial (NCT06413537) testing the oral candidate in 10 adult men with fragile X, ages 18-45. The study, which hasn’t started enrollment, will take on a crossover design, where participants will each receive one dose of SPG601 and one of a placebo, separated by one week, in random order.

The trial, which is expected to start this month, is mainly intended to evaluate the treatment’s effects on clinician- and caregiver-reported impressions of clinical improvements and changes in patients’ response to auditory stimuli. Other clinical measures of effectiveness and safety will be evaluated as secondary goals.