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5
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59
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1
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We are at the most important moment in IDefine’s history. For years, we have diligently prepared, building IDefine into a "Research Ready" organization. Now, we are pivoting to a "Treatment Focused" strategy, and we need your help to seize this amazing opportunity.
Your donation today funds four crucial projects that will start driving our efforts from the bench to the bedside, creating a path to life-changing treatments for Kleefstra syndrome.
The KS Challenge: A Life Defined by Need
Kleefstra syndrome (KS) is a rare genetic disorder caused by a single gene mutation (in EHMT1). For families, KS means a daily reality of significant hardship:
- Profound Communication Barriers: Many children have limited or no functional speech.
- Cognitive and Behavioral Challenges: Significant intellectual disability and challenging behaviors.
- Seizure Burden: Difficult-to-control seizures that require constant management and hospitalization.
Your gift changes this reality. This campaign is about funding the scientific work that will allow us to one day mitigate the challenges associated with this disorder.
Paths to a Treatment
We are launching a three-pronged scientific attack to find a treatment. Funding all three diversifies our approach and maximizes our chance of success.
- Gene Therapy: Develop a therapy to insert a perfect copy of the EHMT1 gene.
- Gene Upregulation: Use cutting-edge technology to "turn up the volume" on the one good copy of the EHMT1 gene our children possess, increasing the amount of missing essential protein.
- Drug Repurposing: Screen existing, FDA-approved drugs to quickly identify compounds that could offer immediate symptom relief
Why Give NOW? This is Our Inflection Point.
We are no longer just building the infrastructure; we are funding the solutions. This is the moment to move from being "Research Ready" to delivering "Treatment Focused" investments.
- Urgency: The scientists are ready to begin work immediately in Q1 2026. Delays in funding mean delays in signing contracts and starting the lab work that leads to a cure.
- Momentum: Success in these initial projects will attract major pharmaceutical partners and unlock millions in follow-on funding.
Please don't wait. Be the catalyst for treatment and help us hit our $500,000 goal today!
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