Empowering Communities to Support ALD Research
Why Cure ALD?
In 2024, the Wiener family discovered that their three-year-old son had ALD after the mother took a genetic carrier screening test during a subsequent pregnancy. Confronting this devastating diagnosis, they quickly realized two critical gaps: first, insufficient funding directed toward research focused on curing ALD or preventing symptom onset; and second, a lack of resources to help newly diagnosed families take meaningful action in the fight against this disease.
1 in 17,000
people are affected with ALD
Young boys face the most devastating form
WE ARE FOCUSED ON A CURE
Cure ALD exclusively funds research aimed at curing ALD or preventing any symptom onset, and by empowering families as active contributors to the cause through both fundraising and direct action that advances treatment possibilities.
Why a Cure?
There is reason for optimism. ALD is caused by mutations in the ABCD1 gene, which affects the body’s ability to create the protein that helps break down very long–chain fatty acids (VLCFAs). Although ALD manifests differently across individuals, understanding the cause allows more researchers to be targeted in their prevention and treatment efforts. Advances in gene therapy offer a promising breakthrough in addressing the consequences of ABCD1 mutation.
At the same time, since ALD is a rare genetic disease, it faces significant challenges in securing research funding due to its small patient population.
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Limited market viability
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Difficulty recruiting enough patients for robust clinical trials
This scarcity of funding impacts the pace of research and the development of new treatments. As a result, funding for ALD research is heavily reliant on donations, patient advocacy groups, and non-profit organizations like Cure ALD. Cure ALD is committed to funding research that directly advances a cure or prevents the onset of ALD symptoms. Your contributions to this effort is vital to ensuring that research for ALD continues, despite the financial hurdles.