As we've reached the midway point of 2023, we want to take a moment to provide you with an update on our progress, share some exciting news, and express our heartfelt gratitude for your support in our mission to cure FOXG1 syndrome and related neurological disorders.
First and foremost, we are immensely proud to report that we are well on track to achieve our goals for the year. This progress would not have been possible without the continued dedication and encouragement from supporters like you.
On the research front, we have made significant strides in our gene therapy and antisense programs. Encouragingly, both of these initiatives have yielded promising results in our preclinical models. Additionally, we have identified key pathways that can be targeted by FDA-approved drugs, offering potential avenues to alleviate major symptoms. The screening of several hundred FDA-approved drugs on our zebrafish models has opened up new possibilities for future treatments.
A major breakthrough in our efforts is a new publication from our FOXG1 Patient Registry, which is the most comprehensive data on FOXG1 syndrome in patients to date. This invaluable information is instrumental in ensuring our preparedness for clinical trials, bringing us closer to the day when effective treatments can be made accessible to those in need.
As we continue our relentless work with utmost efficiency and diligence, we cannot ignore the heartache we have experienced in losing too many FOXG1 children this year and over the years. Their memory drives us forward, reinforcing the critical nature of our mission to make a difference in the lives of those affected by this devastating condition.
It is with the deepest conviction that we firmly believe, together, we can provide every FOXG1 child in the world with a life free from suffering. Your support has been an integral part of this collective effort, and for that, we extend our heartfelt gratitude.
Thank you for standing with us on this challenging, but immensely rewarding journey. We are confident that brighter days lie ahead for all those affected by FOXG1 syndrome.
Congratulations and thank you to our Director of Clinical Research, Elli Brimble and our esteemed team of Key Opinion Leaders. This publication marks a major milestone in the work to deeply understand the phenotypes of patients with FOXG1 syndrome so that we can target the most effective therapies.
A special thank you to every FOXG1 parent and caregiver who registered their child and who lives this challenging life every day.
Read FOXG1 KEY PAPERS HERE | Register Your FOXG1 Child HERE
After learning about the success of the new anti-epilepsy drug, Fintepla in other rare disease epilepsy clinical trials, we began working with NYU and Zogenix (Now UCB) to launch a clinical trial for FOXG1 patients. It was important for us to make this an open trial with a remote option so that FOXG1 patients do not have to travel to NYC to participate.
This will be the first of many clinical trials designed to help all of our children live easier, healthier lives.
Learn More HERE.
Our lead scientists and parents to Yuna, Dr. Soo-Kyung Lee and Dr. Jae Lee, who run the The FOXG1 Center of Excellence at UB have been awarded $1.5 million from The Simons Foundation Autism Research Initiative for our FOXG1 gene therapy work!
Did you know that FOXG1 is an autism-related gene and the work we're doing has a great potential to help solve autism?
We are extremely lucky to have the Lee's working nonstop to find a cure for FOXG1 syndrome. The combination of being brilliant scientists as well as FOXG1 parents make their determination and devotion to finding a cure unparalleled in the field.
This grant is a great help towards our $20M fundraising goal for our promising FOXG1 gene therapy program.
Continue reading the July newsletter HERE