Accelerating the Development of Life-Changing Treatments for ARID1B-RD
The Foundation for ARID1B Research (FAR) was started in 2021 by a group of parents who share the heartbreak of receiving a diagnosis of ARID1B-Related Disorder (ARID1B-RD) for their child, often after a lengthy and stressful diagnostic journey.
We learned that our children may never talk, that their intellectual functioning would be impaired, that they are at high risk for debilitating seizures and other medical complexities, and that they would most likely never be independent. We found ourselves on an emotional roller coaster ride, filled with fear and anxiety about our children's future. We tried to navigate a new world of rehabilitation therapies and children's hospitals while educating ourselves about this rare condition.
We were also told that there is no cure. But, we were determined to leave no stone unturned in searching for a better life for our loved ones and all those affected by ARID1B-RD. Our hope has sparked as we discovered the incredible advancements in clinical genetics and the enormous achievements of other parent-led rare childhood disorders organizations in directing the best science to develop treatments for their children. We met world-leading clinicians, scientists, biopharmaceutical companies, and leaders from the rare disease community who confirmed that impactful therapeutics for ARID1B-RD are within reach.
It became clear that we must lead the effort to support the development of treatments. And so, FAR came to life and began to take root. Since its founding, we have created a network of top experts from academia, research institutions, and pharmaceutical and biotech companies. These experts guided us in the development of a roadmap to therapeutics that directs FAR’s efforts. We have started to support research on potential treatments and are now
working to secure funding that will enable us to expand translational ARID1B
research and the development of promising treatment options.
FAR is dedicated to pursuing an aggressively funded research agenda aided by the world's leading scientific, biomedical, and clinical experts.
We aim to bring the most promising treatment strategies into medical practice as fast as possible. A board of parents runs FAR, and we are actively working to establish the scientific advisory board.
2021
ARID1B-related disorder (ARID1B-RD) is a rare genetic disorder that causes significant development delays and disabilities. Children and adults with ARID1B-RD typically have intellectual disability of variable degree, and may also have autistic features, seizures, delays or (near) absence of speech, as well as daily living and motor impairments. Most individuals with ARID1B-RD require continuous care throughout their lifespan and often struggle to live independently. Changes (variants) in the ARID1B (AT-Rich Interaction Domain 1B) gene are the most common cause of Coffin-Siris syndrome.
At this point, there is no treatment for our loved ones, but the breathtaking advances in the application of novel clinical modalities give realistic hope that impactful treatments are within reach.
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The Foundation for ARID1B Research (FAR) is committed to finding impactful treatments for all individuals living with ARID1B-RD across the world, regardless of age or genotype.
FAR will explore the utility of the most promising therapeutic modalities to achieve this goal. When a specific modality shows potential in treating ARID1B-RD, the Foundation will work to advance it into clinical trials. To make this possible, FAR will become a hub for translational ARID1B research by forming collaborations with researchers, biopharma companies, and the patients’ community.
Learning of a loved one’s ARID1B-RD diagnosis deeply shakes a family’s world. Diagnosis day often brings a blend of mixed emotions and a flood of questions. It’s a hard path to walk, but we want you to know that you are not alone.
FAR's Parent Survey will allow you to join our community by providing contact information and basic information about your child's diagnosis. Through the survey, you can share with us anything you want including if and how you want to take an active role in FAR's efforts. You can complete the survey here (it takes only 3-4 minutes).