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The drug exists. The children are waiting. Help us fund the preclinical trial.

“Imagine knowing there’s a drug ready in the lab, the science is solid, and the children who need it are right in front of you, progress is stalled for one reason: lack of funding.

What would you do?”

Sponsor Annual Conference

Help us bring together leading scientists, clinicians, biotech partners, and families from around the world. Your financial support strengthens collaboration and drives faster therapeutic progress.

Sponsor

Natural History Study (NHS)

Looking ahead to 2026, CURE GABA‑A plans to prepare for an mRNA clinical trial. In order to prove the therapies effectiveness we must establish a comprehensive Natural History Study that captures the real-world progression and needs of our patient population.

Sponsor

Protein Replacement Therapy

Support our groundbreaking work developing a protein replacement therapy designed to restore healthy GABAAR function. This program has the potential to transform the lives of children with these rare and severe disorders.

Our founder pioneered this approach by successfully dosing her own daughter, who has a Rett Syndrome. Building on that landmark experience, CURE GABA-A is now preparing to launch proof-of-concept studies for GABRA1 and GABRG2, two of the most affected genes in our community.

Sponsor

“Life is genetic Russian roulette, no one knows who will be next. Help us build the cure now, so if it happens to someone you love, the answer will already exist.”

The drug exists. The children are waiting. Help us fund the preclinical trial. That’s the reality for CURE GABA-A families battling GABA-A receptor disorders, a rare and life-threatening category of genetic epilepsies. Some children are trapped in their bodies, bedridden, unable to eat by mouth, and unable to ask for help because they are nonverbal. Others can walk, but are surprised by hundreds of seizures each day. Some seizures are so severe that no medication can stop them, sending families to the ICU again and again. And heartbreakingly, our community has already lost children to this devastating disorder. Help us fund a cure.  

“Together, we can go from drug-in-a-fridge to child-in-the-playground.” – Monica Joanna Elnekaveh

Every dollar moves us closer to human trials that could save lives. This isn’t theory. This is ready. The drug is literally sitting in the fridge. We just need your help to take the next step.  This is for hundreds of children.

At CURE GABA-A, we’ve made extraordinary progress:

    • We have funded a targeted mRNA nano lipid particle drug for GABRA1 & GABRG2
    • We’ve secured a proof of concept contract with Grann Pharmaceuticals
    • We’ve funded and secured the mouse models required to begin testing

  6. But we are missing one thing: the additional funds to test the target mRNA nana lipid particle drug on the mouse model.

“Imagine knowing there’s a drug ready in the lab, the science is solid, and the children who need it are right in front of you, progress is stalled for one reason: lack of funding. What would you do?” – Agustina Fernandez

mRNA Project: $1.3 Million

Where Your Donation Goes:

Pre-clinical trial budget is $380,000.

Every donation helps cover:

    •  General lab & cellular work
    •  Vivarium + mouse testing
    •  Therapeutic creation costs
    •  Scientist salaries

 

      Clinical Development Cost Breakdown $1,000,000:

        • General FDA expenses
        • Clinical Trial Sites
        • Patients Costs
        • Additional IRB Fees
        • Insurance
        • Salaries

      How You Can Help?

      • Donate today — even $10 makes a difference
      • Make a tax-deductible gift before year-end
      • Ask your employer about company gift matching
      • Share this with your community 

      🔬 CURE GABA-A is a registered 501(c)(3) nonprofit EIN: 93-1989277

      Learn more: www.curegabaa.org

      💌 Contact us: info@curegabaa.org

      📍 Based in the U.S., supporting families globally