Session Descriptions
Simons Searchlight Update
Navigate the ecosystem of rare disease research with an expert guide to the Simons Foundation. Learn how the biorepository, Research Match, and SFARI Base work together to fuel discoveries. Plus, get the latest by-the-numbers look at GRI participants, genetic variants, and the vital data shaping our community's future.
Keynote 1: GRISpeaks
This deeply personal and essential session offers a powerful platform for the GRI community to share their authentic stories. Hear directly from individuals living with GRI disorders and their parents as they recount their unique diagnostic journeys, the daily realities of living with GRI, and their hopes for the future. This is an opportunity to gain invaluable insight into the lived experience of GRI disorders, fostering understanding, empathy, and connection within our collective journey.
Breakout Session #1: Living GRI: Behavior Management & Safety
This practical session focuses on the at home realities of managing the complex behavioral landscape associated with GRI Disorders. The session bridges the gap between clinical theory and daily survival, providing families and caregivers with a toolkit for creating a stable, secure environment.
Breakout Session #1: Learning GRI: Rethinking Iglurs (Expert level)
Learn more about the recent scientific discoveries that challenge the fundamental, traditional assumptions of how ionotropic glutamate receptors (iGluRs) behave. These discoveries may have implications for how certain variants in GRI genes that encode iGluRs affect iGluR functioning and lead to specific symptoms.
Keynote 2: Clinical Trials Update; Phase 3 Breakthroughs
This keynote session offers an unprecedented look at the forefront of therapeutic development for GRI Disorders. We bring together executives from three leading biopharma companies currently conducting Phase 3 clinical trials for which GRI Disorder patients are eligible. Hear directly from representatives of GRIN Therapeutics, Lundbeck, and Praxis Precision Medicines as they share critical updates on their pivotal studies. This is a unique opportunity to gain insights into the progress, potential, and next steps for these late-stage trials, representing a significant stride towards new treatments for the GRI community.
Breakout Session #2: Living GRI: Transitioning to Adulthood
Building on the foundation laid during GRICON25, this session serves as a vital continuation of our Adulthood series. As our children grow, the questions shift from early intervention to long-term independence and quality of life. This panel brings together experts and veteran families to navigate the specific complexities of the transition to adulthood.
Breakout Session #2: Learning GRI: ASOs/Genetic Therapies (Intermediate Level)
Leading researchers in GRI Disorders provide an overview of the latest research on gene therapies for treating GRI Disorders in mouse models, including the use of Antisense oligonucleotides (ASOs). These therapies are particularly promising as they are designed to address the root cause of GRI Disorders and significantly improves the lives of affected individuals.
Keynote 3: How to of Hope Part II - Guide to treating and curing GRI Disorders and Census Update
Get an exclusive sneak peek into CureGRIN's soon-to-be-released, groundbreaking "How to Cure GRI Disorders" guide. This pivotal session will highlight the vital contents of this comprehensive new resource, meticulously designed to chart a tangible path forward for the entire GRI community. Discover how this guide goes into specifics for 11 different GRI genes, detailing strategies for diverse genetic variations including haploinsufficiency, gain-of-function, and loss-of-function dominant negative effects. This deep dive into actionable strategies and essential knowledge is invaluable for empowering families, researchers, and clinicians in the urgent quest for effective treatments and cures, and provides critical insights for those in pharmaceuticals and biotech driving therapeutic development.
Breakout Session #3: MAPS: Taking an Active Role in Your Child’s Educational & Life Planning
Oftentimes, parents/caregivers sit at planning tables are unsure of what to ask for or how to participate in a meaningful way. School teams will ask for “parent input,” but what does that really mean? How are you supposed to respond to the request for input? What are they actually asking for? Participants in this workshop will walk away with a practical planning strategy that can be used regardless of the age of your child or what country you live in. This planning strategy, called MAPS (Making Action Plans), will help families and caregivers take on a more active voice in the planning process and empower them in advocating for the best services and goals for their children. Participants will also be provided with possible solutions and action steps for common pitfalls some families may experience in the planning process.
Breakout Session #3: Learning GRI - Functional Analysis Panel (Intermediate Level)
This session is focused on how variants are functionally characterized - for example, as gain-of-function or loss-of-function, and implications for treatment approaches.
Networking Sessions
Find Your Circle - Real-time connection for the GRI community. Join tailored sessions to exchange experiences, bridge the gap between lab and life, and strengthen our collective journey.