How to Support Cure Sanfilippo Foundation

Save Matthew

**12/20/25 UPDATE: The fundraiser goals for BOTH Phase I (Dec 1) and Phase II (early 2026) of the EAP have been met!! You helped our family raise over $831,000 since fundraising for the EAP began at the end of Oct ~ we are so grateful for our community's outpouring of love & generosity, and amazed by how God provided in such a short amount of time!

The work is far from finished though--over the next few weeks we will wait for details on the EAP which is projected to start in Q2 or Q3 of 2026 and may need to fundraise further for clinical administration costs (TBD). Meanwhile, any donations received after 12/05/25 will support these possible EAP costs and/or to other Sanfilippo Type B initiatives (gene therapy, research, etc.) for which there is always a need. Thank you!**

Follow Matthew's journey:

Instagram: Instagram.com/Cure.Matthew

Facebook: Facebook.com/CureMatthew/

We Need Your Help

Most parents imagine a bright future for their children--them growing up and meeting many milestones, going to school, making friends, getting a job one day, getting old... That will not be the case for Matthew unless we can get him a treatment soon. His brain and body are dying, and the condition he has will likely end his life before adulthood. We are fighting for his life and the one thing standing in the way is funding. We need your help to save Matthew.

Our Story

Our son Matthew is a sweet and playful kid, loving his family deeply. He adores his brother Nathan, saves treats for Mommy, requests piggyback rides from Daddy, and prays for his grandparents nightly. His big grin warms every heart, and he can be seen toting around his favorite Baymax pillow everywhere.

Matthew's life began with challenges due to multicystic dysplastic kidney syndrome, and at 2 years old, he started early intervention due to mild expressive speech delay. A few months later, impaired hearing and ear fluid led to genetic testing, from which we expected negative results. We could never have imagined what would follow.

A week later, the genetic results confirmed Sanfilippo Syndrome Type B, a rare, neurodegenerative and terminal disease caused by a missing enzyme needed to recycle waste in the cells. As a result, toxic material builds up in the brain and other organs, leading to progressive damage and eventual early death.

Our hearts were shattered. I cannot explain the unimaginable grief we felt, to know that our sweet and vibrant son who loves to sing, laugh, and play -- will soon lose the ability to think, talk, and move, dying after just a short time in our lives. This was the most difficult and daunting thing we have ever faced.

We have spent the past 1.5 years navigating the world of rare disease -- talking to doctors and researchers, going to appointments and therapies, fundraising, advocating, and searching far and wide for help. Any help.

We quickly learned that Sanfilippo Syndrome Type B has NO cure, NO treatment, and NOTHING accessible in the clinical trial stage. It seemed unlikely Matthew would receive any treatment before irreversible decline set in.

A Window of Opportunity

However, in just the past few weeks, a rare window of opportunity has opened up. For the first-time ever, a potential treatment for Sanfilippo Syndrome Type B is on the horizon and within reach. We realize this is a crucial, God-ordained moment, with a narrow window of opportunity to make a difference.

A company has agreed to create an expanded access program that would allow a number of Sanfilippo Syndrome Type B children to receive treatment before it obtains FDA approval, helping them stay healthy and potentially slowing or even preventing decline.

To move this treatment forward, $6 million total is needed, with $3.8 million required by Dec 1 to take the next critical step.

Our family has decided to step out in faith and partner with other Sanfilippo Type B families to fundraise for this cause -- because this is the only possible option available right now to save Matthew and other Sanfilippo Type B children.

We're asking you to walk with us. While we can't guarantee the outcome for Matthew and other children, we know we cannot miss this. This treatment could pave the way for dozens of children and redefine the future for families affected by this diagnosis. It's a chance to change what's possible for those who come next.

How You Can Help

Pray. Please intercede to God for all the children affected. Pray for wisdom, stamina, and God's provision in this endeavor
Be a voice. Share this page with your community. Text a friend. Post it online. Every share brings us closer to a breakthrough for kids with Sanfilippo Type B. One share could change everything
Give. Please consider donating to support this opportunity -- a gift of any size makes a difference

Follow Our Journey

Instagram: Instagram.com/Cure.Matthew
Facebook: Facebook.com/CureMatthew/

Other ways to donate without fees

Fidelity Charitable:

search for "National MPS Society" or "Cure Sanfilippo Foundation". Include "in honor of Matthew Lau" in the memo

Mail a check to:

National MPS Society

c/o Donations

PO Box 14686 Durham, NC 27709-4686

Include Memo: "in honor of Matthew Lau"

or

Cure Sanfilippo Foundation, P. O Box 6901, Columbia, SC 29260. Include "In honor of Matthew Lau" in the memo line

Don't forget to check if your company has a donation match! You can submit a tax receipt if you donated outside of your company's matching portal.

FAQ

Q: What does the expanded access program look like?

A: The expanded access program aims to provide treatment to 14 kids for 1 year via compassionate use (a pathway for patients with serious conditions to access investigational treatments when no other options are available and they can't join a clinical trial). This buys time for the kids, preventing regression while awaiting FDA approval.

Q: Where do your donations go?

A: Donations pass through the Foundation and Society, then the biotech company, to pay for manufacturing of the treatment. No fees are taken by the Foundation and Society or the biotech company. The manufactured treatment will be used in the expanded access program, with Matthew's potential participation in 2026

Q: Are we fundraising alone, or with others?

A: We're fundraising alongside 16+ families impacted by Sanfilippo Type B, as well as other Sanfilippo types families selflessly jumping on to support

Q: What is the fundraising goal?

A: About $6M with two phases of fundraising: $3.8M by Dec 1, followed by $2.2M by early 2026

Q: What happens if we don't reach our goals by the deadlines?

A: If the program doesn't move forward, all funds will go toward alternative Sanfilippo Type B treatments

Q: How is Matthew doing?

A: Matthew is continuing to develop with his favorite activities including scootering, fetching mail with mommy, baking cookies, and playing with big brother Nathan. But with Sanfilippo Type B, progression is unpredictable - skills like talking can vanish in a matter of weeks. We are racing against time to secure a treatment!

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387 supporters

Morgan Rachal is fundraising

Save Lydia

A parent's worse nightmare is losing their child. Our daughter Lydia is 3 years old. She is so full of life. She loves playing right along side with her big sister. But her little body is dying. Before her disease takes over her body and brain damage starts to occur, we have one chance to change her fate & it has to happen soon. What if money was the one thing that stands in the way of your child's chance to live? We need your help to save our sweet Lydia. **donations are tax deductible**

Our Story

When Lydia was born, we were so excited. Her big sister was so happy to have a forever best friend. Our hearts were so full. She had a perfect birth. The doctor said “she’s perfect”. We were so in love with her. We were so happy to have our new girl to take home with us. 18 months of our lives were just full of happiness. Until April 4, 2024, the day our lives changed forever.

Our sweet Lydia was diagnosed with a rare genetic disorder called Sanfilippo Syndrome type B, which is terminal. Life Expectancy not passed teenage years. Our hearts were shattered and it was a dark place I cannot even explain. How was our child slowly dying and we did not even know it?

In March, my mother sent me a TikTok of a little girl named Olivia with the TikTok account "Saving Liv". This little girl could have been my Lydia's twin. It was a gut punch when I saw the diagnosis of Olivia being a terminal illness. Of a disease I did not even know existed. So I started researching more of the disease, with symptoms that went along with it. I found frequent ear infections, sleep disturbances, distended belly, GI issues, and prominent thick eyebrows, which were all the symptoms Lydia had. The thick eyebrows were something I thought she inherited from my husband. Everyone always said "They look just alike". I knew then when I saw the symptoms, this was what my daughter had. My gut told me so but my heart told me there was absolutely no way (it is 1 in 70,000) that my baby who has met all milestones in her life and to me, was a healthy baby as I thought.

I took my concerns to our pediatrician in our hometown of Natchitoches, Louisiana. I showed him side by side pictures & the TikTok I had seen. He told me "I have never been asked this before, nor have I ever seen a child with it". He told me he would have to go do research himself and come back into the room. He came back into the room and tells me "I cannot come back in here & tell you she does not have it, so I will order you the genetic testing for it, but you will have to do it at another hospital because our town does not have that type of ability to test for it. I want you to assure you that this is very rare, and children come in all the time with issues not sleeping, constipation, and frequent ear infections still after tubes". I was so grateful that he listened to me, ordered us the testing, and did not make me feel crazy. We went to another hospital to have the testing done the very next day. It was a blood test and a urine test.

A week of anxiously waiting to "rule it out".... the following Thursday I pull up my patient portal to see POSITIVE MPS III (Sanfilippo Syndrome). That exact moment was the worst feeling of any day I have ever had on this earth. My heart sunk, I felt like I could not breathe. The instant feeling of "my baby is going to die".

This dark place my husband and I were in last for about 2 weeks. It was then, I was ready to fight for her. I contacted the Cure Sanfilippo Foundation to figure out what I could do as a mother to fight for her and the treatments available for her. There are no current treatments or cure approved by the FDA, only clinical trials. But there is HOPE. The clinical trials have a gene therapy treatment and an enzyme replacement therapy. In Sanfilippo Syndrome, Lydia's body is missing an enzyme that is used to breakdown sugar molecules. Without this enzyme, toxic waste build up in her body. This would explain her distended belly from an enlarged liver. That I thought was just from constipation. As the years go by and more toxic waste build up, it will start affecting her brain, causing damage, leading to her to lose or forget all of her abilities such as walking, talking, and feeding herself, everything she has already learned. This is why it is called Childhood Dementia. She will also eventually endure pain and suffering, and then die. This is 100% fatal.

Luckily, we were able to get diagnosed early because of TikTok and our pediatrician. The regression usually starts around age 3 and 4. But every child with this disease is different. This would mean we have about a year or less before brain damage begins & takes over. Fast forward 18 months after we got the diagnoses, THERE NOW IS A TREATMENT OPTION. We need 6 million to fund a treatment that could be available for her as early as this summer 2026. 3.8 million by December 1, 2025 & the rest by spring 2026. This is how you can help us Save Our Lydia. So one day, when my 6 year old daughter, Heidi, is able to understand, I will not have to explain to her that her little sister is going to die. The words I never want to hear is "I don't want my sister to die". After the diagnosis, that is the moment I am dreading. She is the best big sister to her. Her sister deserves a chance at life.

As a mother, feeling helpless and knowing this disease is out of my control.. the only thing I know to do is fight for her. Fighting for her in this case is making awareness of this disease, sharing her story, fundraising, and in hopes her story will reach the world. Hoping the world will help save her. We pray everyday for this. We will never stop fighting for her. Every dollar counts. We need a miracle and this world of a community could be our miracle. Along with all the other children and families suffering with this disease and heartbreak.

Our goal is to raise $6 million dollars to help fund treatments and give our Lydia a chance at growing up, a chance at life. We can't just sit back and accept that she is going to die with knowing there are treatments out there. Previous clinical trials done for Sanfilippo Syndrome have shown real promise. The treatments WORK. Our Lydia needs treatment!! This campaign for her LIFE is as urgent as it gets. It's now or never for Lydia. This is our last chance to save her life before brain damage begins. 3.8 million by December 1st.

This is truly our only hope. Her story has already reached millions. People Magazine, VT, Daily News, & News stations. Help us keep sharing her story. In hopes it will reach a celebrity or millionaire that will be these children's spokesperson and help fund for them. They deserve a chance at this life. A parent's love is like no other. If you are a parent, you understand.

There are three things we are asking you to do. A campaign like this can grow quickly if supported and if shared with great mass. We can reach this goal if it goes viral.

1. Please donate what you are able. All donations go to the 501c3 nonprofit Cure Sanfilippo Foundation (Tax ID: 46-4322131) to fund a clinical trial and are tax deductible.

2. Please take a few minutes and share our GoFundMe everywhere you can. It’s really easy to do. Just click the “Share” button and send it everywhere you can. Please help this reach the right people. I know if they see it, this can happen.

3. Please leverage any avenue you have to make this go viral.

Thank you for making it this far in our story. I hope you are able to help save our girl.

-Morgan & Kirk

Other ways to donate:

-- By check: Cure Sanfilippo Foundation, PO Box 6901, Columbia, SC 29260. Include “Save Lydia” in the memo.

-- Donate via Venmo, ACH, digital wallet or digital wallet, go to https://give.curesanfilippofoundation.org/save_lydia

-- Make a recurring donation, go to https://give.curesanfilippofoundation.org/give/630751/#!/donation/checkout?recurring=1

-- By stock gift, email [email redacted] for details.

-- TikTok: https://www.tiktok.com/@hopeforlydia

-- Instagram: https://www.instagram.com/hopeforlydia/

-- Facebook: https://www.facebook.com/profile.php?id=61561244030850

For media

For additional information and photography for the media, please go to http://CureSanfilippoFoundation.org/Save_Lydia

Credits

Special thanks to Joe Lane of Anointed Media LLC (https://www.anointed.media) for lending his tremendous videography, video edit, music and audio editing skills, and passion to this project.

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24.3K supporters

Erin Stoop is fundraising

Saving Liv

Our daughter Olivia just turned three. She is energetic and loving. And she’s dying. We have one chance to change her fate and it has to happen soon. We need your help to save Liv’s life.

When Olivia was born, we were so excited! We couldn’t wait to start showing her the world and watching her experience life. Dance lessons, teaching her guitar, and baking together. All those things you envision for your child.

When Olivia was born, she passed all the newborn screening tests and was given a clean bill of health. However, in her first two years, she constantly had recurring ear and sinus infections and frequent loose stools. We pressed Liv’s pediatrician for answers at her two-year-old check up and began seeing a series of specialists, hoping for an answer. We were so scared the answer would be some type of chronic condition that she would have to live with her entire life. Little did we know that something even worse lay ahead.

In March 2022, our world came crashing down. Olivia was diagnosed with Sanfilippo Syndrome Type B, a genetic degenerative brain disorder in children caused by a single missing enzyme. It’s the worst disease you’ve probably never heard of. It’s like Alzheimer’s, but in children. It’s 100% fatal and there currently is no FDA-approved treatment or cure.

Because Liv lacks this single enzyme, toxic cellular waste material is building up in her brain every second and damaging it. As the degeneration rapidly progresses, Liv will stop walking, stop talking, stop being able to feed herself. She’ll develop movement disorders and seizures, suffer severe dementia, endure a lot of pain and suffering, and then she’ll die. All in the coming years.

Today, Liv loves playing waitress, helping take care of her baby brother Liam, and singing with us during bedtime snuggles. Without a cure, that will all go away and Liv will pass away in her teens.

But there is hope! There are promising forms of treatment that are already being investigated for Sanfilippo Syndrome. They just need funding to continue and reach the clinical trial stage.

Current promising research underway that could lead to clinical trials includes gene therapy, substrate reduction, and drugs already-approved for other diseases that could also help children with Sanfilippo. All of these focus on lessening the build up of the toxic cellular waste in children’s brains and bodies, which reduces neuroinflammation in the brain and prevents development of Sanfilippo’s many disease symptoms.

It just needs funding.

Large pharmaceutical companies have little interest in rare diseases like Sanfilippo because they don’t see ways to make a profit. Angel investors and federal grants are incredibly difficult to obtain. Which leaves funding the research that could save our children to parents, patient-led organizations (like Cure Sanfilippo Foundation), and kind-hearted people willing to donate.

We need your help to fund research so a clinical trial can happen.

Our goal is to raise $1 million dollars to fund the research that could lead to a clinical trial and give Liv a chance at growing up, a chance at life. We can’t just sit back and accept that she’s going to fade away and die, not when there is something we can do to try to save her.

This is as urgent as it gets. It’s Liv’s only chance.

There is no guarantee Olivia would be accepted into a trial. But it’s the only chance. And without funding for research and clinical trials, her future is certain. But we have to try. This is our only hope.

A parent’s love for their child is like no other. If you are a parent or simply love kids, you understand. Please, please help save our daughter.

There are three things we’re asking you to do. A campaign like this can grow quickly with your support. You are the key! We have faith and pray every day that your kindness, generosity, and compassion will help us make the difference for Liv.

1. Please donate what you are able. All donations go directly to the 501(c)3 nonprofit Cure Sanfilippo Foundation (Tax ID: 46-4322131) to fund research for Sanfilippo Type B and are tax deductible.

2. Most importantly, please share this page with everyone you know via social media and email by clicking share button or typing in SavingLiv.com.

3. Please leverage any avenue you have to make this go viral. Send leads or tips to us by clicking the Contact button at the bottom of this page.

Please help us give Olivia a chance at life. It has to be now.

Our deepest thanks. Words can’t even express how incredibly grateful we are.

Erin and Tyler Stoop

Follow the latest:

-- On Instagram: https://www.instagram.com/saving_liv/

-- On TikTok: @saving_liv

-- On Facebook: https://www.facebook.com/profile.php?id=100088622245570

Donations are tax-deductible.

Other ways to donate:

-- At Liv's campaign on Cure Sanfilippo website, donate via Venmo, PayPal, ACH, digital wallet at this site.

-- To make a recurring donation (monthly, weekly, quarterly, or annually), click here.

-- www.CureSanfilippoFoundation.org (click “Donate” and put “Saving Liv” in the dedication)

-- By check: Cure Sanfilippo Foundation, PO Box 6901, Columbia, SC 29260. Include “Saving Liv” in the memo.

-- By stock gift, message us and ask for details by clicking contact button at the bottom of this page.

-- By cryptocurrency at www.CureSanfilippoFoundation.org/crypto

Special thanks to the artists, who graciously dedicated their time and talent:

Video footage by Alex Sharon, Pilgrim Visual, LLC

Video editing by Benjamin Von Wong

Photography by Traci Stoop of Stoop Images, the Stoop Family, and Vanessa Wyler of Vanessa Wyler Photography

Original music by KeithTim Anderson

Audio mastering by Andrew Kesler

Information for the media: https://www.curesanfilippofoundation.org/SavingLiv

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WHERE IS YOUR MONEY GOING?

Every dollar goes to Cure Sanfilippo Foundation, a 501(c)(3) nonprofit organization which funds research that can lead to a clinical trial for Sanfilippo Syndrome Type B that could give Olivia a chance at life. Donations are tax deductible. 100% of the funds raised here will go toward the research. We are currently racing against time to fund this potentially life-saving research that could lead to a clinical trial. It will help Liv and so many other children have a chance at life, today and for generations to come. ***Any donations received above the goal will go directly to Cure Sanfilippo Foundation new research projects in need of funding***

WHY SO MUCH MONEY?

Getting a clinical trial up and running can cost millions of dollars. In rare diseases, large pharmaceutical companies have little interest because they don’t see ways to make a profit. The truth is that it is left up to parents, patient-led organizations, and donors to drive and fund the research for treatments and a cure. This will help fund research that could lead to a clinical trial that can possibly help Olivia and other children with Sanfilippo. Raising this amount of money may seem daunting, but it has been done before and research and clinical trials have happened as a result. It can for Liv too.

TIME IS OF THE ESSENCE

Sanfilippo Syndrome causes children’s cognitive and physical abilities to be completely taken away quickly once they begin regressing. In time, children regress back to an infantile state, likely never to return. This rapidly-progressive neurodegenerative disease will soon start taking over completely. Olivia is 3 years old. We don't want to lose our daughter. This must happen now.

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3.9K supporters

Rachel Wood is fundraising

Save Oren

Last year, we learned that our 4 year old son Oren was dying, that there was no cure, no treatment, no fight to be won. But you can help him have a chance to live. *donations are tax deductible*

Our Story

Oren is an outgoing and joyful 6 year old boy who loves playing with his younger brother and sister and his cousins. He is expressive, has a contagious smile and laugh, and loves others unconditionally. When he was born, he had several minor complications which required surgery to correct. But according to doctors, the surgeries went well and there was nothing to be worried about going forward.

However, when Oren was 2 years old, we began to notice challenges: difficulty learning to talk, developmental delays, chronic bowel issues, and trouble sleeping. We immediately started him in speech therapy and enrolled him in a preschool program to support his development.

Despite early intervention, constant specialist visits, and our efforts at home, something still wasn't right. Not only was Oren not improving, his condition was worsening.

After two years of doctor visits, research, and questioning, we still had no answers, no clear path forward to help our son. Not until a family friend, who is a nurse, saw a video on TikTok with a child who had symptoms strikingly similar to Oren come across her feed. The child had Sanfilippo Syndrome. The more we learned, the more we knew this was what Oren was facing. We contacted our pediatrician asking for a referral to a geneticist so he could be tested for Sanfilippo Syndrome. We were quickly referred to the nearest children's hospital, where testing began. `

On July 31st, 2024, we learned that our 4 year old son Oren tested positive for MPS IIIB -Sanfilippo Syndrome Type B- a terminal disease with no cure and no available treatment. Commonly referred to as "Childhood Alzheimer's," Sanfilippo will take everything from him. He will lose his ability to speak the few precious words he knows, Oren will lose his ability to chase after his siblings and cousins, he will eventually forget all those he loves most. And then he will die.

But now there is HOPE.

Just over a year after his diagnosis, there is an actual treatment option available for children with Sanfilippo Type B. This is truly a medical breakthrough in the world of Sanfilippo/MPS III. We believe that you can help our Oren get his miracle. For months, we laid by Oren's bed, praying and crying out for his healing and for a cure, and we promised we would do whatever was in our power to fight for him, for a cure, for better quality of life. We didn't know if we had the faith it would take to believe for a miracle, but now there is a path that could lead to one. Our hope is that bringing awareness to this rare childhood disease could help Oren and other beautiful Sanfilippo children have the opportunity to share their lives with us a little longer.

Whether you can give financially or not, you can play a role in changing history.

How You Can Help

1. Pray 

Pray for the children impacted by Sanfilippo B. We believe that God is opening doors and that he is not finished with this story yet. Hope is rising. We have seen God moving in every step of our journey with Oren.

2. Give 

A gift of any size makes a difference.

Your gift doesn’t just help our family — it helps create access for many children battling this disease.

Other ways to donate:

--Donate on the Cure Sanfilippo Website (transaction fees apply; donors may opt to cover them): https://give.curesanfilippofoundation.org/campaign/653569/donate

--For gifts of $500 or more, checks may be made payable to:

Cure Sanfilippo Foundation

P.O. Box 6901

Columbia, SC 29260

3. Share

Awareness is key.

Share our GoFundMe. Just click the "share" button. Tell your community. Text it to a friend.

One share could be the one that changes everything.

Follow Us

Facebook: Save Oren (https://www.facebook.com/share/1DMYPnKtHk/?mibextid=wwXlfr)

Cure Sanfilippo Website: https://curesff.org/oren

Instagram: @saveoren

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142 supporters

Christin Ulrich is fundraising

Saving Veda

I'm Christin, Mom to Veda. When Veda was born, Jericho and I thought we had our fairytale life. That this was the beginning of our happily ever after. We never expected that our entire world would turn upside down in a matter of years. And that one TikTok video would change our lives forever.

When Veda was born, she seemed like a completely normal baby, aside from some breathing issues right at birth. Those first days, weeks, and years were just bliss. Counting fingers and toes, and dreaming about our family’s future.

Veda is one of those “big personalities,” filled with vibrance and joy. She is always smiling and loves to be goofy. And, I’ll admit, she’s definitely Daddy’s little girl, through and through. She has this second sense for when it’s time for Jericho to come home from work and she always waits for him to arrive. As soon as he walks in the door, she’s running to him, giving him hugs and kisses. The two of them together are something special.

But Veda began falling behind in meeting some developmental milestones and we became concerned. Like every parent, we went to the pediatrician in search of answers for her delayed speech and chronic ear and upper respiratory infections. Sadly, we were met with repeated assurances of “She’s fine.” and “She’ll catch up,” from doctors, despite having a growing list of concerns. We had to push relentlessly to be referred to specialists. We spent months trekking to appointments with specialists, who ran test after test, but they didn’t have answers either.

My mother’s intuition was telling me there was something wrong, something causing all these concerning symptoms in Veda. Despite all the assurances from doctors, I knew there was something going on and I had to find the answer.

I never imagined what I was going to find and how I would find it. Especially since I wasn’t even looking.

One night, I’m scrolling TikTok in bed after Jericho had already fallen asleep. I see a video of a mom talking about her daughter, Haidyn, who looks like Veda’s twin. In the video Haidyn’s mom is talking about how Haidyn has a disease called Sanfilippo Syndrome and so many of the symptoms sounded like what we were seeing in Veda.The realization Sanfilippo Syndrome could be the reason for all Veda’s symptoms hit me like a punch.

I immediately Googled “Sanfilippo Syndrome” and, as I started to read, the world came crashing down.

Sanfilippo Syndrome is like Alzheimer’s, but in children. It is a terminal, degenerative disease that causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, dementia, and then die usually in their teen years. It’s caused by a single gene defect which leads to lack of a necessary enzyme in the body. This lack of enzyme results in the build-up of toxic storage in every cell, and is especially harmful to the brain. This build-up causes a cascade of detrimental effects and ultimately death. Currently there is no FDA-approved treatment or cure for Sanfilippo Syndrome.

The fear racing through me was like nothing I had ever felt. I was so scared that I woke up Jericho, hoping he could convince me this couldn’t be true. We both prayed it wasn’t Sanfilippo. We couldn’t face the idea that our daughter has peaked in her development, that she would lose every single skill she’s ever gained, and that she will die in the coming years after years of extreme suffering.

In September 2022, genetic testing confirmed that Veda has Sanfilippo Syndrome. All those years of searching, begging doctors for help, and we finally had an answer. An answer that was worse than anything we could have ever imagined. We were completely crushed.

We spent the first few weeks after her diagnosis stricken with grief. But we decided we had to fight. We had to do our best, like any parent would, to save our child. We reached out to Cure Sanfilippo Foundation, for opportunities that might give Veda a chance.

But there is hope! A clinical trial for Sanfilippo Syndrome is in the preclinical stages (drug production, drug safety testing, efficacy testing, IND application process, etc.). With enough funding, this will become a clinical trial that begins treating multiple children. And we need it to start as soon as possible. Not just for Veda, but for all children with Sanfilippo.

It just needs funding, and a lot! Clinical trials for rare diseases are enormously expensive.

Large pharmaceutical companies often have little interest in rare diseases like Sanfilippo because there isn’t enough of a business incentive. Angel investors and federal grants are incredibly difficult to obtain. Which often leaves funding the research that could save our children to parent-led organizations like Cure Sanfilippo Foundation. Cure Sanfilippo Foundation works with the community of Sanfilippo families to rally kind-hearted people (like you!) willing to donate.

Your donation will help make the clinical trial happen!

Our goal is to raise $500,000 to help fund the clinical trial. Being part of a clinical trial will give Veda a chance at growing up, a chance at life. We can’t just sit back and accept that she’s going to fade away and die, not when there is something we can do to try to save her.

This is as urgent as it gets. It’s Veda’s only chance.

There is no guarantee Veda would be accepted into the trial. That decision is up to the clinicians running the trial. But it’s the only chance. We have to try. This is our only hope. And without funding for research and clinical trials, her future is certain: she will lose all of her abilities, suffer, and pass away, likely in her early teens. All right before our eyes.

We’ve had to let go of a lot of dreams, of things we thought our future held. We have one dream now, one desperate hope: Saving Veda.

Please, please help save our daughter.

There are three things we’re asking you to do. A campaign like this can grow quickly with your support. You are the key!

Please donate what you are able. All donations here go directly to the 501(c)3 nonprofit Cure Sanfilippo Foundation (Tax ID: 46-4322131) to help fund this clinical trial and are tax deductible.
Just as important, please share SavingVeda.com with everyone you know via social media and email so it keeps spreading.
Please leverage any avenue you have to make this go viral. Click 'contact' below to share any leads you might have with us.

Please help us give Veda a chance at life. It has to be now.

Our deepest thanks. We are so moved and humbled by your support,

Christin, Jericho, Elise (Veda’s older sister), and Veda