Rare disease funding is limited. Join Cure ALD’s Rare Disease Day fundraising efforts to support the research and development of new ALD treatments. All money raised will be directly donated to carefully vetted research projects that align with Cure ALD's mission to find a cure or preventative treatment. Take a look at the incredible projects below.

Why now?

X-linked Adrenoleukodystrophy (ALD) affects 1 in 17,000 people. ALD is a rare genetic disorder caused by mutations in the ABCD1 gene, leading to the accumulation of very long chain fatty acids (VLFCAs) that damage the brain, spinal cord, or adrenal glands with varying severity. Young boys face the most devastating form—childhood cerebral ALD—which can be fatal without early intervention. While treatment options exist for some manifestations, there is no cure or preventative treatments. Since there is limited funding for rare disease research, funding for ALD research is heavily reliant on donations. Your donation or fundraising effort is vital to ensuring that research for ALD continues. Find out more about Cure ALD.

Cure ALD Research Network

Your gift, your choice! When you donate, you can direct your dollars to the general ALD research fund (split between both projects) or give to a specific project you're passionate about. We believe you should have a say in the impact you make.

Project 1: Assessing Nervonic Acid's Potential to Protect Adrenal Function in ALD

Project Lead: Keith Van Haren, MD — Stanford School of Medicine

This study will explore whether nervonic acid, a natural fatty acid, can prevent adrenal failure in a genetic mouse model of ALD. Dr. Kartha’s laboratory at the University of Minnesota has already shown that nervonic acid lowers the harmful fats (VLCFAs) that damage adrenal cells. Now the Van Haren Lab and Kartha Lab will work together to test whether giving nervonic acid to ALD mice can protect their adrenal glands before damage occurs.

The challenge with adrenal failure in ALD has always been that doctors can only treat it after it happens, with lifelong steroid medications. The Van Haren lab will measure whether nervonic acid can lower harmful fat buildup in adrenal tissue, prevent cellular damage, and reduce stress hormones. The intent for this is to help design clinical trials of nervonic acid as the first preventive treatment for adrenal insufficiency in ALD boys. Learn more about this project →

Project 2: Safer Gene Therapy for ALD

Project Lead: Alex LeNail, PhD — George Church Lab, Wyss Institute at Harvard University

Alex is exploring a new way to treat Adrenoleukodystrophy (ALD) using gene therapy. The goal is to safely deliver a working copy of the ABCD1 gene to the brain and nervous system—the areas most affected by ALD.

The challenge with treating ALD has always been getting therapeutic treatments into the brain, which is protected by something called the blood-brain barrier. He’s testing specially designed delivery vehicles that may be able to cross this barrier more safely and effectively. Learn more about this project →

Introducing Cure ALD Ambassador

Sydney (Zmrzel) Luke, known to WWE fans as Maxxine Dupri

Read more about Zmrzel Family here.

For this campaign, Sydney is bringing some serious firepower:

Everyone who donates by Feb 28 will be entered into a raffle for a worn and autographed t-shirt.
Everyone who donates $100 or more will be entered to win an autographed gear — one Sydney actually wore inside a WWE ring. (Yes, really.)
Sydney is also kicking off the campaign with a $5,000 donation match — meaning every dollar donated this month is doubled, up to $5,000

Interested in Fundraising?

Follow these steps to set up your own Rare Disease Day Fundraising page.

Profile picture - have a photo of yourself ready to upload to the page.
Fundraising goal - every dollar has the power to make an impact, so there is no goal too small or big. It’s important to think about the level of impact that is attainable within the timeframe you’ve set. Generally $5,000 is a realistic target for an online fundraiser.
Your story - Cure ALD and ALD in general will likely be new to your audience, so it’s important to explain why you’ve chosen to partner with Cure ALD - to raise funds exclusively aimed at supporting research to cure ALD.
Create your page - select the “+Create a Page” button above in the Fundraisers section to register as a fundraiser and get your own page started using the information from #1-3.
Promote your page - share your page with friends and family via email, text, or social media.
- Use this Email & Text Toolkit and view sample messages that you can copy and edit for personal use.
- Use this Social Media Toolkit (via Canva) to create images for your social media posts.

💙Thank you for fundraising with Cure ALD. We are stronger together! #HelpCureALD

Rare Disease Day - Feb 28, 2026

Fundraisers