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Help Advance Treatments for NIPBL-Related Cornelia de Lange Syndrome

Your gift funds the next critical steps: understanding the biology of NIPBL variants, rigorously testing promising treatment candidates, and building the patient data infrastructure needed for clinical studies.

Every dollar matched, up to $500K

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Your donation is tax-deductible

Our plan: move science toward treatments

The NIPBL Therapeutics Foundation is raising $3.2 million to accelerate the development of treatments for NIPBL-related Cornelia de Lange syndrome.

Double your gift. Help raise our first $1 million.

Our founding donors will match every dollar you give, up to $500,000 in total matching funds. Together, $500,000 from supporters and $500,000 from our founding donors will fund the first $1 million of our research plan.

What your gift makes possible

1

Understand the biology. Define how NIPBL variants disrupt chromatin organization and gene regulation, and use these insights to prioritize therapeutic targets.

2

Test promising treatments. Expand our drug-repurposing study and develop cell and animal models to evaluate which candidates should advance.

3

Close critical gaps in patient data. Track how the condition changes over time to help show the FDA whether future treatments work. That work starts now.

4

Build the team that drives progress. Establish our core operating team to lead scientific work, coordinate clinical research, and strengthen patient data.

The work has already begun

Our first NIPBL drug-repurposing study identified promising candidates. We’re developing patient-derived induced pluripotent stem cells (iPSCs), which can become specialized cells, including neurons, to study NIPBL biology and test treatment candidates in a lab.

This next phase will evaluate whether these early leads can restore biological function and show potential for therapeutic benefit.

Founded by a family. We bring personal urgency and commitment to rigorous science.

INTERESTED IN SUPPORTING RESEARCH SPECIFICALLY FOR YUVAAN'S VARIANT?

Please contact Shivika at shivika@nipbl.org to discuss arrangements for your gift.

Other ways to give

To give stock, recommend a donor-advised fund grant, or donate by bank transfer or check, contact Shivika at shivika@nipbl.org.

About your donation

The NIPBL Therapeutics Foundation is fiscally sponsored by Rare Village Foundation, a qualified 501(c)(3) nonprofit. Donations are received by Rare Village Foundation in support of our work and are tax-deductible to the extent permitted by law.

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