Join The Registry

By sharing your information, you help us continue to grow the largest global GABAAR (GABA-A Receptor) patient community, strengthen natural history data, and accelerate the development of real treatments. What began with one little girl’s journey has grown into a global effort to move science forward for all families affected by GABAAR variants.

Major Milestone: Partnership for mRNA Therapy Proof-of-Concept.

“In a historic milestone for genetic medicine, the first-ever mRNA therapy was successfully administered to a patient, marking a new era for disorders like GABAARs.” Press Release

In August 2025, CURE GABA-A reached a historic milestone by announcing a major new partnership with Grann Pharmaceuticals to develop nanolipid particle (LNP) protein replacement therapies. This collaboration follows the successful completion of the initial safety regimen for RTT-1 (ELEANOR), marking a critical step forward for rare neurodevelopmental disorders. Press Release

Key Achievements:

Strategic Partnership: Formalized agreement to lead preclinical animal studies targeting GABAAR variants (specifically GABRA1 and GABRG2).
mRNA Innovation: Utilizing an innovative therapeutic platform to restore healthy GABA-A receptor function.
Global Network: This milestone connects our patient community directly with industry experts to move from “drug-in-a-fridge” to real-world clinical readiness.

This breakthrough represents our commitment to turning research into treatment and bringing hope to every family in our global registry.

Your Information Here

Join Us / Register

First Name (Caregiver)

Last Name (Caregiver)

First Name (Patient)

Last Name (Patient)

Email Address

Phone Number

Street Address

Street Address2

City/Town

State/Province/Region

ZIP/Postal Code

Country

Your Connection to the Patient

GABA-A Receptor Diagnosis

Instruction Video

Variant Mutation ex. (p.Q239E C715C>G)

List All Genetic Mutations

Symptoms

Share Your Story

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By Checking Here You Give Us Permission to Feature and Publish Your Story on our website

Consent to Data Collection and Use I voluntarily provide my personal, medical, and/or genetic information to CURE GABA-A Variants (“CGA”). I understand that this information will be used for community support, education, advocacy, and internal program development. I acknowledge that the CGA community registry is not a clinical trial and is not intended to conduct regulated human subjects research. I understand that my information will not be used for research intended to produce generalizable scientific knowledge or shared with external researchers, clinicians, or industry partners without appropriate ethical approvals (including IRB approval, where required) and additional consent. I understand that my information will be kept confidential, will not be sold, and may be used in a de-identified or aggregated manner to support CGA’s mission. I understand that participation is voluntary and that I may request removal of my information at any time by contacting CGA.

Consent to Be Contacted for Research Platform Enrollment I understand that after completing registration with CURE GABA-A Variants (“CGA”), I may be invited to enroll in external research platforms, including Matrix and Citizen Health, through CURE GABA-A–affiliated portals. I understand that these platforms operate under Institutional Review Board (IRB)-approved protocols and that participation is voluntary and separate from the CGA community registry. I consent to being contacted by CGA with information and access to enroll in these platforms. I understand that any participation will require additional informed consent directly through the respective platform.

Why Parents Choose to Share Their Data

If you’re here, it’s because you’re searching for answers, options, and hope. We understand that sharing your child’s information is a big decision.

1️⃣ How your child’s data helps create treatments

Without patient data, therapies cannot move forward.

Your child’s information helps us:

Build the natural history required for clinical trials
Show researchers and industry that this community is real and ready
Accelerate the development of targeted therapies

This is how we move from waiting to action.

2️⃣ This work is already happening

We are not starting from zero.

The first mRNA therapy has already been given
Active mouse studies are underway right now
Clinical readiness is being built in real time

Your participation connects directly to real progress.

3️⃣ You are not alone

Rare disease can feel isolating. It doesn’t have to be.

By joining, you become part of:

The largest global GABA-A community
A network of families, clinicians, and researchers
A movement built by parents, for parents

4️⃣ What this could mean for your child

Participation may help:

Identify opportunities for future studies or trials
Connect you to emerging research
Keep you informed as treatments develop

5️⃣ Your privacy and control

We take your trust seriously.

Your data is used to advance research and clinical readiness
All personal and medical information is kept strictly confidential, with privacy controls in place to limit access to authorized individuals only.

6️⃣ Why this matters

Every family who shares their story strengthens the science.

What began as a fight for one child has become a pathway for many.

Your child’s data has the power to shape the future of treatment. Your voice and participation are important. Your story is important and your data is important. Please share it.