FAMILIAL DYSAUTONOMIA FOUNDATION

The FD Gene Therapy and Drug Development Fund

For the first time, the science to treat the root cause of Familial Dysautonomia is within reach. Three genetic therapy programs are underway right now. Help us see them through.

$2.0M $1.0M $0
$507,000
raised of our $2,000,000 goal · 25% of our goal

Where your gift goes

FD is caused by a mutation in the ELP1 gene that starves sensory and autonomic neurons of a protein they need to survive. These three programs each target that root cause.

1. Gene replacement therapy

An AAV vector delivers a healthy copy of the ELP1 gene. In FD mouse models it raised ELP1 protein and protected neurons. We have human patients in trial for almost two years. Results are positive and we are expanding trials in 2026. In partnership with NYU Langone.

2. Retinal therapy for vision loss

Targeting the progressive optic neuropathy that steals sight from FD patients in their teens and twenties. Getting FDA approval for initial human testing. Working with leading researchers around the world. This therapy uses existing proven technology for delivering gene therapy directly to the patient’s eye.

3. Splice-correcting small molecule

A drug that corrects the ELP1 splicing defect to restore full-length, functional protein throughout the body. Tests have begun on FD mouse models. Will move to human testing in 2027. This is a partnership with renowned researchers at Massachusetts General Hospital.

Check Donations:
Familial Dysautonomia Foundation
Gene Therapy Fund
719 2nd Ave #505
New York, NY 10016

Wire Transfer or DAF Donations:
Email the FD office at [email protected]
or call 212-279-1066

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Photos by Rick Guidotti, POSITIVE EXPOSURE
EIN 13-6145280

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Medical Disclosure
The FD Foundation does not offer medical advice. We always recommend that you contact the Dysautonomia Center (link here) or your personal physician.