OUR STORY

 

SIMON needs HELP! A new treatment option has become AVAILABLE.

Simon has Sanfilippo Syndrome. It’s a terminal, neurodegenerative rare disease, and causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, and then die, often before the second decade of life.

Because of its neurodegenerative nature and multi-system impact, Sanfilippo Syndrome is often called “childhood Alzheimer’s” or “childhood dementia.”

Children with Sanfilippo Syndrome are born with a single genetic defect, a single change in their DNA, that causes their bodies to lack a necessary enzyme.

Because of the lack of enzyme, their bodies are unable to breakdown heparan sulfate, a natural cellular waste. Instead, their bodies and particularly brains become clogged with toxic levels, which causes a cascade of detrimental effects and ultimately death.

Currently there is no FDA-approved treatment or cure. BUT a phase 2 Enzyme Replacement Therapy (ERT) clinical trial has been showing high efficacy, safety, and effectiveness. So, the Sanfilippo community has been presented with an opportunity to have an Expanded Access enzyme replacement treatment for some children with Sanfilippo Type B, Simon’s type!

Simon could be one of just 14 children in the world to receive life-changing treatment for Sanfilippo Syndrome Type B — a devastating rare disease.

This could give Simon and others more time, more hope, and a chance to fight back.

But there’s one big challenge:

The community must raise $4 million by Thanksgiving to secure the manufacturing slot for this treatment — or the opportunity disappears.

A few weeks to change lives. A few weeks to make hope real.

This isn’t a typical fundraising ask. It’s about buying time — literally — for children whose futures depend on access to this treatment.

Cure Sanfilippo Foundation and the National MPS Society are working together to make it happen, but they can’t do it alone.

As of 11/7/2025, we parents of kids with Sanfilippo Syndrome Type B along with the Foundation and Society have already raised ~$1.3M for our Type B kids’ chances to benefit from this Expanded Access opportunity!

With your help, we can keep the momentum going to reach our goal.

How You Can Help Right Now:

Time is short, but hope is powerful. Together, we can help Simon and the 13 other children reach the gold at the end of the rainbow.

Much love for your continued support,

Alina and Jeremy (Simon’s parents)

#HelpSimon #ExpandedAccess #RareDisease #EnzymeReplacementTherapy #SanfilippoSyndrome #hope

 

Follow the latest:

 

-- On Facebook: https://Facebook.com/HelpSimonNow

 

-- On TikTok: https://TikTok.com/@Simons_Shot

 

-- On Instagram: https://Instagram.com/HelpSimon

 

**Donations are tax deductible.**

 

Tax ID Number: 46-4322131

 

Other ways to donate:

-- By Venmo: Click here to donate via Venmo Comment "Expanded Access - Help Simon"

-- By Foundation's website: Go to https://CureSanfilippoFoundation.org (click “Donate” and put “Expanded Access - Help Simon” in the dedication)

 

-- By check: Cure Sanfilippo Foundation, PO Box 6901, Columbia, SC 29260. (Include “Expanded Access - Help Simon” in the memo.)

 

-- By ApplePay, MicrosoftPay, or ACH: Go to https://CureSanfilippoFoundation.org/Donate and select your preferred payment form at the bottom of the screen.

 

-- To be a recurring donor (monthly, quarterly, weekly, etc.): https://give.curesanfilippofoundation.org/HelpSimonMonthly

 

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

 

Video by Paul Shirley (https://www.paullikescameras.com/)

 

Video Editing by Ben Von Wong (https://www.vonwong.com/)

 

Original music by KeithTim Anderson (https://keithtimanderson.com/) ISRC: QZELS2190001

 

Audio mastering by Andrew Kesler (https://andrewkesler.com/) 

 

Photography by Amy Mikler Kenyon (https://amymikler.com/)

 

 

Information for the media: https://CureSanfilippoFoundation.org/HelpSimon

 

 

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WHERE IS YOUR MONEY GOING?

 

Every dollar goes to Cure Sanfilippo Foundation, a 501(c)(3) nonprofit organization helping to secure the manufacturing slot for this treatment — or the opportunity disappears. Donations may be tax deductible. NOTE: $382,856 of the HelpSimon.com funds has already been spent on the CRL01 and stem cell projects, so all funds donated above this dollar amount are allotted for the Expanded Access Program and will go toward our collective goal of $4M by Thanksgiving 2025. ***If the goal is not reached in time, the donations will be reserved for future efforts to save the lives of children with Sanfilippo Type B.***