We’re excited to share an update on our Drug Repurposing Observational Study!
Thank you for your patience as this has taken longer than anticipated to launch. The Drug Repurposing Observational Study is a major step forward in our mission to find treatments for individuals living with BBSOAS.
As we discussed at our conference in April, thanks to a grant from the NR2F1 Foundation, Dr. Chow and his amazing team screened over 1,600 existing medications that are already approved for use in the U.S. and Europe. From this extensive search, more than 30 compounds showed promising improvements in our fruit fly models of BBSOAS. After careful consideration of factors like safety, accessibility, and the specific needs of our community, they have narrowed this down to TWO drugs. While there are still 6-8 weeks of work ahead before we can dive into the details, please know that this project is a top priority for the NR2F1 Foundation and we are working as quickly as we can to launch the study.
Need a recap of the Drug Repurposing Project?
➡️ Watch an interview with Research Chair, Melissa Thelen, here.
➡️ Watch a discussion with Dr. Chow reviewing the details of the study here.
What can you do to get ready to participate in the study?
We expect to launch this study in the next 6 to 8 weeks, and in order to participate, it is essential that you join the Patient Registry. If you are registering for the first time, create a profile, complete the surveys, and add your genetic report. Already registered? Just go in and retake your annual surveys.
3 Easy Steps to Take Part in the Patient Registry:
**or **
to the NR2F1 Patient Registry (Matrix platform)
Complete all surveys
in your dashboard — more may appear based on your answers
Upload your child’s genetic report
(crucial for research accuracy)
🔎 Tip: Register yourself as the caregiver first, then add your child as the patient.
Need help? Email Amanda.Reiser@nr2f1.org. She can help give guidance and reset passwords!
We’re committed to keeping you updated as we reach important milestones along the way. Thank you so much for your ongoing support and partnership in our journey toward better treatment options for those with BBSOAS. Together, we can drive real change.