Cure GABA-A | What do we need to know about Gain of Function GABAAR
2024-05-28 11:00 - 12:00 (America/Los_Angeles) | FL
What do we need to know about Gain of Function GABAA receptor variants?

Understanding Gain of Function GABAA Receptor Variants
GABAA Receptor Variants | Research Insights Session | Dr. Lidong Liu
Join us for an enlightening session with Dr. Lidong Liu, Senior Research Scientist at the University of British Columbia’s Department of Neurology and the DM Centre for Brain Health.
Dr. Liu, in collaboration with Dr. Yu Tian Wang, focuses on the molecular mechanisms of synaptic function and plasticity, particularly under conditions of health and disease. This session will describe GABAA receptor variants and their implications for epilepsy.
These groundbreaking insights are crucial for developing better treatments for conditions like epilepsy. Dr. Liu’s work highlights the dynamic interplay between neurotransmitter systems and offers new avenues for therapeutic intervention.
Event Details:

Date: 5/28/2024
Time: 11am PDT

If you cannot attend in person, don’t worry—the session will be recorded and available on our YouTube channel.
Join us to learn from Dr. Liu’s expertise and contribute to our shared mission of improving the lives of patients with GABAA receptor variants.
Project Overview:

GABAA Receptor Study: This project focuses on understanding GABAA receptors.
Initial Discovery: The first GABRA1 variant (A322D) was identified in epilepsy patients about 20 years ago.
Recent Work: Recently, more variants have been characterized, and some repurposed drugs have been tested on these variants based on requests from doctors and patients’ families.

Key Discoveries:

Glutamate and GABAA Receptor Crosstalk:
Significance: There is an important interaction between glutamate and GABAA receptors.
Mechanism: Glutamate can bind to and enhance GABAA receptors to balance out the overexcitation caused by glutamate receptors.
Research Model: Mice with mutations in the glutamate binding site of GABAA receptors showed increased neuronal excitability, seizure susceptibility, and autism-like behaviors.
Netrin-1’s Role:
Function: Netrin-1, a molecule that guides axons, is secreted during high neuronal activity.
Effect: It binds to GABAA receptors and increases their function, helping to balance synaptic activity by enhancing the conductance of these channels.

Cure GABA-A | Dr. Ortiz Community Update Meeting
2024-05-29 10:00 - 11:00 | FL
We invite you to a special presentation that will NOT be recorded. Dr. Ortiz will discuss three key points for our community.

We invite you to a special presentation that will NOT be recorded. Dr. Ortiz will discuss three key points at our upcoming Cure GABA-A meeting for patients and caregivers:

1. The preliminary results of the antiseizure medication survey completed by our community.
(https://filadelfia-science.dk/surveys/?s=TEX9JXNK3CPWTNA4)

2. An explanation of the Sleep Disturbance Scale for our children.

3. The GABAAR Movement Disorders clinical video proposal featuring the MyDystonia electronic diary.

We hope to see you all there.

Cure GABA-A | Artificial Intelligence (AI)-driven Drug Repurposing for Rare
2024-06-27 13:00 - 14:00 | FL
Aleksandra Foksinska, Program Manager, and Alexandria Howard, Case Analyst at UAB Hugh Kaul Precision Medicine Institute, research AI-driven

Aleksandra Foksinska and Alexandria Howard will both discuss Artificial Intelligence (AI)-driven Drug Repurposing for Rare Disease.
Aleksandra Foksinska is the Program Manager at the UAB Hugh Kaul Precision Medicine Institute. Her background is in Molecular Biology (BS) and Biomedical Sciences (MS) from the University of Alabama at Birmingham. Aleksandra’s research interests include identifying drug repurposing candidates for rare genetic disorders using AI. Aleksandra also serves as a project coordinator for the UAB Center for Precision Animal Modeling, as well as a committee member for the Undiagnosed Diseases Network Therapeutic Matching Committee.
Alexandria Howard is a Case Analyst at the UAB Hugh Kaul Precision Medicine Institute. She is currently approaching her senior year at the University of Alabama at Birmingham with a double major in Genetics/Genomic Sciences and Neuroscience and a minor in Chemistry. Alexandria’s research interests include investigating various rare genetic disorders and identifying drug repurposing candidates using AI.

Cure GABA-A | Genotype-phenotype correlation&precision treatment of epilesy
2024-06-28 10:00 - 11:00 | FL
The research focuses on genetic epilepsy, aiming to identify severe epilepsy genotypes and develop precision therapies.

The current landscape shows increasing insights into human genetics, yet the translation of genetic data into actionable patient care concepts lags behind. This gap is influenced by two primary challenges: the variability in clinical outcomes associated with different genetic variants within a single gene, complicating treatment decisions, and the empirical nature of current epilepsy treatments due to the absence of targeted therapies for specific genetic changes.
Our specific focus is on the genetic LOF variant D120N in the GABRB3 gene’s beta-3 subunit, known for severe forms of developmental and epileptic encephalopathy (DEE), such as Lennox Gastaut Syndrome (LGS). This variant’s location in the gene’s extracellular region suggests a correlation with clinical severity based on functional domains. However, understanding which genetic variants correlate with mild or severe presentations remains a challenge, impacting prognostication and treatment strategies. Furthermore, the pathophysiology and underlying cellular mechanisms associated with these genetic variants require further investigation.
Through this research, I aim to contribute to the advancement of personalized medicine, ultimately improving outcomes for children affected by genetic epilepsy.

CURE GABA-A | Grann Pharmaceuticals mRNA Protein Replacement for GABAARs
2025-09-26 13:00 - 14:30 | FL
First glimpse at mRNA protein replacement for GABAAR disorders. Join CURE GABA-A + Grann Pharma live Q&A this Friday at 1PM PST.

CURE GABA-A | Grann Pharmaceuticals: mRNA Protein Replacement for GABAAR Disorders

Date & Time
📅 Friday, September 26th 2025🕐 1:00 PM PST | 4:00 PM EST
Location
Online Event (Zoom link provided upon registration)
Description
🚨 STOP WHAT YOU’RE DOING 🚨
This is the moment everything we’ve worked for comes down to. We started CURE GABA-A with one mission: to get pre clinical data for treatments relating to GABAAR disorders as quickly and safely as possible. And now, we are finally at the beginning.
Grann Pharmaceuticals has developed an mRNA-based protein replacement therapy for GABRA1 and GABRG2 variants. This therapy is already produced and ready for preclinical testing. With the right support, we can bring it to the FDA and into clinical trials by the end of 2026.
But science alone isn’t enough. We need the community to band together now, fundraising, donating, and spreading the word, to make this therapy a reality for children and families.
Join us for a live Q&A with Tommy Temple, CEO of Grann Pharmaceuticals, as he explains:

What mRNA protein replacement therapy is
Why it could be the first treatment for GABAAR disorders
The FDA pathway and clinical trial timeline
How YOU can make the difference to bring this to clinic

💙 Together we can move from hope to treatment.

Agenda

Welcome from CURE GABA-A
Presentation by Tommy Temple (CEO, Grann Pharmaceuticals)
Live Q&A session
Call to Action: How to Fundraise and Donate

Tickets
🎟️ Free – Registration required.Donations encouraged: https://www.facebook.com/donate/4300256346872932/
or GoFundMe: https://www.gofundme.com/f/lifesaving-gene-therapy-drug

Call to Action
✨ Every donation moves us closer to the first-ever GABAAR clinical trial.✨ Every fundraiser brings hope to families waiting for answers.
👉 Register now, join the movement, and help us make history.

2026 CURE GABA-A Family & Scientific Conference
2026-11-05 - 2026-11-07 | Hyatt Regency Orlando, Hyatt Regency Orlando 9801 International Drive, Orlando, FL, 32819, United States
2026
CURE GABA-A Family & Scientific
Conference

November 5-7th • 8:00 am
HYATT REGENCY ORLANDO, FLORIDA

Register to Attend

Sponsor the Conference

Two days of scientific presentations, family programming, GABA-A Receptor research updates, and global collaboration.

2023 Roundtable

2024 Conference

2025 European Conference

2025 Conference

Countdown

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What, When and Where

What:
2026 CURE GABA-A Family & Scientific Conference

When:

Disney Day | November 5th
Family Meeting | November 6th
Scientific Meeting | November 7th
Where:
Hyatt Regency Orlando9801 International DriveOrlando, FL 32819

Featured Talks & Speakers

Full Schedule Available soon

What to Expect
Discover the leading voices shaping the future of GABAAR research.
Our 2026 conference features presentations from global experts in GABAAR science, neuroscience, clinical care, gene therapy, and translational research.
Attendees will hear the latest updates on GABAAR science, therapeutic development, natural history studies, iPSC research, animal models, and real-world clinical insights.

SCIENTIFIC PROGRAM

FAMILY PROGRAM

BIO SAMPLE COLLECTION

2026 Conference Highlights

GABA-A receptor biology and disease mechanisms
Natural history studies and the importance of clinical trial readiness
Patient registries and data collection
Biomarkers and clinical outcome measures
Preclinical models and translational research
Gene therapy and therapeutic development
Clinical trial readiness and future therapeutic pathways
Global collaboration across the GABA-A community

Venue
Our venue will be the Hyatt Regency Orlando Conference Center

Hotel Room Block (Across the street)
Rosen Centre Hotel
Address: 9840 International Drive, Orlando, Florida 32819
Official program dates: Wednesday, November 4, 2026 through Sunday, November 8, 2026
Conference dates: November 6–7, 2026
Disneyland/Disney family day: November 5, 2026, with more details coming soon
Suggested arrival: November 4 for families attending the Disney day
Suggested checkout: November 7 or November 8, depending on travel plans
Group name: CURE GABA-A
Room block name: CURE GABA-A – Room Block
Total rooms blocked: 160 room nights
Room block pattern: * November 4: 20 rooms * November 5: 50 rooms * November 6: 50 rooms * November 7: 40 rooms
Group room rate: $169 per night for single/double occupancy
Additional person fee: $20 per person
Taxes/fees: Room rate is exclusive of taxes and fees, currently including 12.5% state/local taxes and a 1% OCCCD fund
Children: No charge for children ages 17 and under when using existing bedding
Booking deadline: Monday, October 5, 2026 at 5:00 PM EST
After the deadline: Reservations will be accepted based on availability and may be at the higher of the group rate or the hotel’s available rate at that time
Booking by phone: Guests may call 1-800-204-7234 or 407-996-9842 and ask for the CURE GABA-A group rate
Online booking link: Coming soon
Deposit/payment: Individual guests are responsible for their own room, tax, and incidental charges
Reservation guarantee: Rooms must be secured with a credit card, debit card, cash, check, or money order
Cancellation policy: Individual guestroom cancellations must be made at least 5 days before arrival to avoid forfeiting one night’s room and tax
Shortened stay fee: Changes to departure date after check-in may result in a one-time $75 administrative fee, unless due to a documented family emergency
Check-in: After 3:00 PMCheck-out: 11:00 AMWi-Fi: Complimentary basic Wi-Fi is included in guestrooms and public areas, excluding meeting/convention areasResort fee: No resort feeParking: Discounted overnight self-parking rate of $15 per night for attendees during program datesAccessible/ADA rooms: Guests needing accessible rooms or auxiliary aids should request them directly with the hotel when booking
Important hotel note: Guests should confirm they are booking at Rosen Centre Hotel on International Drive, as there are multiple Rosen hotels in the area
Source: Rosen Centre Hotel room block agreement for CURE GABA-A.
Travel Planning
To help families manage costs, we encourage booking airfare as early as July. Flights to Orlando tend to increase in price as summer approaches, so early planning can make a big difference.

On-Site Bio sample Collection

At the 2026 CURE GABA-A Conference, families will have the opportunity to take part in on-site bio sample collection through COMBINEDBrain, our national research and bio collection partner.

Who Can Participate
Families of individuals with confirmed or suspected GABAAR variants are welcome to take part. Samples may include saliva, blood, or other non-invasive collections depending on the research protocol.

Why It Matters
Every sample helps researchers better understand GABAAR variants and how they affect development, seizures, expression, and brain function. These samples accelerate the development of new treatments, including mRNA therapy, protein replacement, drug screening, and biomarker discovery.
Your contribution moves research forward in a tangible, measurable way.

What COMBINEDBrain Does
COMBINEDBrain is a leading research consortium focused on severe neurodevelopmental disorders. They work with top scientists and hospitals to process, store, and analyze biosamples so data can be used responsibly and effectively across multiple research programs. Their work ensures that every sample contributes to real progress.

How to Sign Up
We will release a sign-up link closer to the event. Families will be able to select a time slot that works best for their schedule.
Spaces may be limited, so we encourage early registration once sign-ups open.

Bio Collection Registry

For Families
Learn the latest science in a supportive environment
Connect with families from around the world
Access resources and variant-specific insights
Participate in biosample programs that accelerate research
Meet the scientists who are actively working on GABAAR therapeutics

Researchers & Clinicians
Present your work to a global, engaged audience
Access a rare and concentrated dataset across GABA-A receptor variants
Engage directly with families and community leaders
Collaborate with global labs

For Sponsors
Align with high-impact rare disease research
Gain visibility among families, scientists, and clinicians
Support the advancement of GABAAR therapeutics
Reinforce your corporate commitment to neurological innovation

Our Sponsors

Thanking our previous years sponsors

Register Now!

Ticket Info

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Have questions? We’re here to help.Email: info@curegabaa.org
30765 Pacific Coast Hwy Suite 366 Malibu, CA 90265 United States
EIN: 93-1989277
