Weekly Updates! - May 23, 2025

Weekly Updates! - May 23, 2025

Hello. I hope everyone has had a great week. I have a few updates I’d like to share:

• Recap: Gene Therapy Considerations in TBRS presentation

• • • Earlier this morning (Friday, May 23), Dr. Harrison Gabel presented on the latest advancements in TBRS gene therapy research!
      • Following a groundbreaking article on an individualized gene therapy that saved a child with a rare disease, we received a lot of questions about the potential of gene therapy for Tatton Brown Rahman Syndrome (TBRS).
      • To help answer these questions, we asked Dr. Harrison Gabel for an update on his preclinical work toward gene therapy in TBRS.
      • There was a great turn out, with over 59 live attendees!
    • Dr. Gabel’s presentation provided great scientific information into where gene therapy treatment for TBRS stands.
      • If you missed this presentation, the recording can be found on the TBRS Community private Facebook page

• Join Us for a Special Talk on Hematology & Oncology in TBRS - with Dr. Rachel Rau

• • We’re excited to welcome Dr. Rachel Rau for a special presentation on June 9 at 5 PM EST, where she’ll discuss hematology and oncology considerations for individuals with Tatton Brown Rahman Syndrome (TBRS).
  • This session is a valuable opportunity for families and patients to learn from an expert in the field and better understand the latest insights related to blood and cancer-related health concerns in TBRS.
    • Don't miss this chance to ask questions and stay informed—register here and join us live!
    • If you are unable to attend, please sign up - a recording will be sent to families who are unable to attend!

• SAVE THE DATE: 2026 TBRS Summit

• • The TBRS Summit is planned for early 2026!
    • WHEN: Weekend of March 21, 2026
    • WHERE: Morgan’s Wonderland Camp in San Antonio, TX, USA
  • The TBRS Summit is where the magic happens—families, researchers, and clinicians come together to connect, learn, and push forward the future of TBRS care and research.
  • Stay tuned for more details, including registration and agenda updates!

Thanks! Please reach out to me with any questions at kit@tbrsyndrome.org. Jill and I will be at a conference next week, but I’ll have more updates on Friday, June 6. Happy weekend!Kit Church, CARE Manager