FOXG1 Research Foundation

The FRF team is hitting the conference circuit in 2025. As we advance toward clinical trials for the FOXG1 AAV9 gene therapy, we will be meetings with the experts in the area of clinical operations at the SCOPE conference in Orlando. If you’ll be at SCOPE, please let us know and say hello! About Scope: Clinical trials, digital health, and clinical research are essential for advancing medical knowledge, improving patient care, and developing new treatments and therapies for the patients who need them. Execution of this vital work requires collaboration, innovation, and strategic decision-making. Now in its 16th year of fostering these joint efforts to be inclusive of all stakeholders, SCOPE Summit 2025 will take place February 3-6, 2025, in Orlando, FL. https://lnkd.in/e_xbfAX

Japan, Taiwan, Russia, Pakistan, Israel, Poland, France, Italy, Spain, Brazil, Canada and all around the United States. Parents from all over the world flew to Fort Lauderdale, Florida for our FOXG1 Parents Conference November 15-17. There are no words adequate enough to describe how meaningful this weekend was. FOXG1 children and adults, with their parents and siblings joined leading FOXG1 clinicians, scientists, rare disease leaders, and donors to learn - among many things- about the FOXG1 gene therapy that we are on track to bringing to human clinical trials by 2026. Without Pharma stepping in to help us get treatments to our children, we're doing this on our own and our goal is accelerated approvals. While I was on stage in Florida, Josie was in an ambulance without me. She's ok now, but this suffering has to stop - and we know gene therapy is our best chance to give our kids a healthy life. Our preclinical data is incredibly promising—groundbreaking in fact. And our work has far-reaching implications as we're disrupting the model for rare disease drug development - cutting the cost and time in more than half. Josie turned 13 this past Saturday. Please consider clicking this link below and giving her a birthday gift donation towards a treatment that can drastically improve her life. https://lnkd.in/edaEnTdQ Thank you for supporting in any way. We take Crytpo too! https://lnkd.in/eEvFmVQG If there is anyone you know that would consider a major gift, please connect us so I can present our work. Stay tuned for the entire conference on our FOXG1 Research Foundation YouTube channel. Nasha Fitter Effie Parks Soo-Kyung Lee Terry Pirovolakis Ryan Phillips Brianna Fleming Elli Brimble Angela Van Wingerden, DES

Last week, our Chief Clinical Data Officer, Elli Brimble was invited to speak at the invite-only Chan Zuckerberg Initiative Science in Society event. Elli presented the innovative work the FOXG1 Research Foundation has done around utilizing patient data from our registry and Citizen Health to speed up drug development for our gene therapy program. We’re so proud to be at the forefront of innovation in rare disease research! #FOGX1 #GeneTherapy #PatientData #CZI #ScienceInSociety #RareDiseaseResearch

Today's article in Bloomberg (featuring the cutest 12-year-old girl I know) offers an accurate insight into the unfortunate economic landscape for promising gene therapies for rare diseases. It underscores the critical role of philanthropy. There is substantial reward in saving human lives, but not for Wall Street. As Jorge Conde from Andreessen Horowitz aptly states, "It's unfortunate, because the promise here is substantial." Yes, it is substantial, and as a parent-led rare disease patient organization, we must raise the funds to bring life-changing treatments to our children. However, we also need the government to step in—because these treatments won't just help our children live healthier lives, they will also lower healthcare costs by alleviating the economic burden of the 30 million people in the U.S. alone who live with a rare disease. Parents simply won't give up on programs that can save our kids lives, which is why we are the exact groups that government should support. Yesterday I had to announce to our community that a beautiful 17-year-old FOXG1 girl passed away on Sunday. A few hours later, another FOXG1 dad wrote to me to let me know his son is currently fighting for his life at St. Louis Children's. Yes, parent-led patient organizations will do whatever it takes to drive these treatments to children. It might not be lucrative for Wall Street, but the ROI is our children's lives. FOXG1 Research Foundation FOXG1 Research Center Nasha Fitter Ryan Phillips Amber N. Freed Terry Pirovolakis Gerry Smith #genetherapy https://lnkd.in/giVPbemM

A monumental moment for FOXG1 research! Watch as Robin Schulze, (Dean of College of Arts and Sciences at UB), Nicole Johnson, Nasha Fitter, Dr. Soo-Kyung Lee, Tom Horton, and Venu Govindaraju (Vice President for Research and Economic Development at UB) officially open the FOXG1 Research Center at the University at Buffalo. Here’s to hope and love! #foxg1researchcenter #foxg1syndrome #foxg1research

We are thrilled to announce that the opening ceremony and science symposium will be live-streamed, and we’d love for you to join us virtually!

Big day for the FOXG1 community! We hit a major milestone today - meeting with some of the most well known key opinion leaders Eric Marsh from Children's Hospital of Philadelphia, Pam Ventola from Yale University and Elizabeth Berry-Kravis from Rush University Medical Center to decide on what we will measure in our upcoming gene therapy trial. Big thanks to all our caregivers who participate in our NHS on Citizen Health that have helped to create our retrospective study. #curefoxg1

An important milestone for a rare disease is to establish the correct incidence and prevalence rate. We worked with GeneScape to perform a systematic review and meta-analysis and found that the prevalence of FOXG1 patients per 100,000 children is 0.6–2.2, resulting in up to 1600 patients in the United States. Read the pre-print here: https://lnkd.in/gGq_6h6Q #foxg1 #foxg1syndrome #genescape #raredisease

Seven years ago, Labor Day weekend a group of FOXG1 parents came together with a shared purpose: to find a cure for FOXG1 syndrome. From this meeting, the FOXG1 Research Foundation was born. Our co-founder and CEO reflects here:

Join us for the opening ceremony of the FOXG1 Research Center at the University at Buffalo. ✂️ A science symposium will follow the opening ceremony when scientists from the FOXG1 Research Center will present their work and the FOXG1 Research Foundation will give an overview of the research and development for FOXG1 syndrome. Note: this event is in addition to the upcoming FOXG1 Syndrome Parents Conference in November! Event Details: → Tuesday, September 24 at 9am est. → The Buffalo Room at the University at Buffalo - 10 Capen, North Campus → RSVP by September 12th - https://lnkd.in/eBnfjvd5 *All attending parents must be registered in the FOXG1 Research patient registry. #foxg1syndrome #universityatbuffalo #foxg1research