From the very beginning, our mission has been deeply personal. Born from love, desperation, and an unshakable determination, what once felt like an impossible dream is now becoming real. We refused to accept that nothing could be done, and held fast to the belief that children diagnosed with INAD/PLAN deserve more than just hope.

Thanks to groundbreaking scientific progress, the strength of our growing community, and the momentum we’ve built together, we now stand at the brink of the next chapter. Our preclinical studies are completed, safety and toxicology data collected and preparations for next steps are underway; we’re no longer just working on a miracle, we’re manufacturing our miracle and we need your help to get it across the finish line. Thanks to a VERY generous donation of $750k from Rimma’s parents, Inna & Ivan Makarov, we’re just $450k away from fully funding Manufacturing.

At the center of this effort are the more than 5,000 children around the world whose lives hang in the balance. Children like Rimma, Ariya, Adrien, Zaidee, and Rex, each one deserving of a better future. INAD/PLAN is a cruel disease that robs children of their abilities — crawling, speaking, eating, even smiling — often before their second birthday. There is no cure, and time is their greatest enemy. Most won’t even reach their 10th birthday.

Help us raise the funds needed as we go from “Working on a Miracle” to “Manufacturing our Miracle”!

The Miracle Makers

Because INAD is so rare, it doesn’t get the attention or funding it desperately needs. The big drug companies? They’re not interested, it’s just not profitable for them. This is a heartbreaking reality for families watching their children lose abilities day by day – a nightmare no parent should have to endure.

So what do we do? We take action.

Through the Working on a Miracle campaign, the INADcure Foundation, together with parents, scientists, and supporters, has been building something extraordinary. We’re not just hoping for a miracle; we’re actively working toward it.

Now, we’re entering the next bold chapter: Manufacturing Our Miracle. That means taking all the science, momentum, and community support we’ve built and putting it into action to bring a gene therapy treatment to life. And, we’re doing it together!

After years of fruitless door-knocking and making our case to more researchers and biotech companies than we can count, we were compelled to make a decision. Either we continue to wait for someone else to do it, or we do it ourselves.” - Leena Panwala, Ariya’s mom and Founder of INADcure

The Miracle

We’ve already raised $3 million toward our $5 million goal, proof that so many believe in this miracle right along with us. But we’re not just dreaming anymore. We’re manufacturing our miracle, transforming that belief into real progress. Every single donation, no matter the size, brings us closer to making this life-altering treatment a reality for the children who need it now.

Be One of Our Million!

How amazing it would be if we could get one million people to each donate just $7. Donate $7 today and Be One of Our Million!

5 Lattes

Instead of grabbing a latte on your way to work each day this week, donate $25 towards our Miracle – funding a gene therapy trial.

Stream Instead

Skip going to see a movie this month (stream one instead) and use that money to donate to our Miracle – getting us close to a Gene Therapy trial!

Work on a Miracle!

Together, we can make our Miracle a reality for all those suffering from PLA2G6-Associated Neurodegeneration (PLAN)!

How YOU Can
Be A Part of Our Miracle

We mean it when we say your help is needed! So whether you’re touched by INAD/PLAN personally, a supportive friend or neighbor, or simply someone who cares and believes in miracles, here’s how you can help make a miracle happen:

Donate: Every gift helps us move forward.

Fundraise: Get involved by setting up a fundraising page or joining a team. We’ve got plenty of support to guide you along the way.

Have Questions? Read our FAQ

The Science Behind the Miracle

Science and miracles: an oxymoron? We beg to differ! When we mix a whole lot of hope from our INAD/PLAN community with cutting-edge research, we have a winning formula for a modern-day miracle! The INADcure Foundation is working with top scientists, labs, and medical institutions to make a gene therapy for INAD. Gene therapy involves delivering a working copy of the PLA2G6 gene into cells of the nervous system, particularly those in the brain. Gene therapy could correct the expression of the PLA2G6 gene to restore enzyme function. This correction may prevent some INAD symptoms and slow or stop the progression of the disease.

We have already funded preclinical studies in an INAD mouse model to determine the potential for therapeutic efficacy. The gene therapy treatment extended the lifespan of the mice. This gave us ‘proof of concept,’ which forms the basis for seeking FDA approval. We are now starting dosing studies at Jackson Laboratories, which will determine the right amount to give, and toxicology studies, in partnership with Nationwide Children’s Hospital, to assess safety. After completing these studies, an Investigational New Drug Application (IND) will be submitted to the Food and Drug Administration (FDA) for approval to conduct a clinical trial.

Expenses

$ 0

Income

$ 0

Pre-Clinical Studies and Manufacturing Expenses To-Date (updated 5/31/2025)

Gene Therapy Program Income
(Updated 5/31/2025)

Miracle in
the Making

A Financial Snapshot

Follow how each dollar contributes to advancing our gene therapy program!