Donate to Help Fight for Simon's Life

Hi! My name is Alina. And, our 7-year-old son, Simon, has Sanfilippo Syndrome. It’s a terminal, neurodegenerative rare disease. It causes children to lose all the skills they’ve gained, suffer seizures and movement disorders, experience pain and suffering, and then die, often as children.

Because of its neurodegenerative nature and multi-system impact, Sanfilippo Syndrome is often called “childhood Alzheimer’s” or “childhood dementia.” Right now, there is no FDA-approved treatment or cure.

We learned about our son’s horrible disease when he was two years old. So many of you have supported us ever since we were devastated by this news. Thank you!

When he was three, we persevered in getting Simon into a gene replacement therapy clinical trial. We are forever grateful for this opportunity to improve the quality and length of Simon’s life. It seems it allowed Simon to maintain his skills for three years longer than he would have otherwise. But by six years of age, he began losing his expressive language. So, it seemed as if the benefits of gene therapy had begun to fade.

Now at seven years old, Simon has damage to his brain's white matter, progressive scoliosis, osteonecrosis of his femoral heads, progressive hearing loss, and only a handful of spontaneous utterances remaining - ALL a result of Sanfilippo Syndrome.

But we continue to fight for his life! We recently hired Everlum Bio Rare Disease Research Lab to run an amenability study on Simon. The researchers learned that they could use Simon’s OWN skin cells to create fibroblasts for use in testing the thousands of currently FDA-approved small molecule drugs for therapeutic potential in Simon's condition - Sanfilippo Syndrome. It will cost $50,000 to run the Small Molecule Drug Repurposing Program.

As Simon’s primary caregiver, I am not earning an income. (To learn why it's extremely difficult to be employed when caring for a medically complex child, please watch the Unseen documentary.) So, I humbly ask for your support. Whether you’re new to our story or have long been a supporter, I ask for your help.

Please donate today, if you’re able - $5, $50, $500, $5,000 – it all adds up!

Many of you have supported us in the past, time and time again. You’ve followed our journey with Sanfilippo Syndrome, held space for us through difficult times, and celebrated our joyous moments. You’ve made donations to Cure Sanfilippo Foundation in honor of Simon. You’ve shared Simon’s story with others in order to help us spread awareness about this horrible condition. Thank you!

Also, please share our UPDATED story with family, friends, and colleagues via social media, email, text, and good old-fashioned in-person communication.

It’s now or never for our sweet Simon. The deterioration of Simon’s body is happening all day, every day. So, we aim to find a timely treatment for Simon that will slow the progression of his disease. All with the hope that advances in research will result in a cure that Simon will be alive to receive.

Thank you again for donating and sharing our updated story! We have the potential to find an effective treatment for Simon and other kids around the world with Sanfilippo Syndrome Type B (~4,500 children).

Much love, Alina (Simon’s Mama)

For the latest:

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

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

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