In 2022, Margaret and Bridget were diagnosed with Sanfilippo Syndrome, a neurodegenerative disease that is like Alzheimer's in children. They will slowly lose all the skills they once had.

Currently, there is no FDA-approved treatment or cure for Sanfilippo Syndrome.

But there is hope.

It’s real-and it could change everything for children with Sanfilippo Syndrome 3B. But we need to move fast.

The Sanfilippo patient community has been offered a unique opportunity by a drug manufacturing company to receive enzyme replacement therapy through an Expanded Access pathway. This is not the typical way for Expanded Access to be rolled out; however, given the extreme unmet need, we are grateful for any prospect to access treatment. This opportunity requires the patient families along with the related Sanfilippo foundations to secure the funding.

There are many details that remain unknown, including the criteria for children to be selected for the Expanded Access, the site locations, the exact timing, and much more. Eligibility and enrollment will be determined by doctors and researchers working with the pharmaceutical company.

Potentially, this will supply up to 14 children with one year of enzyme treatment. The path beyond that is uncertain. The hope is that the therapy will become FDA approved during this time and patients would then be able to be covered by insurance to receive the approved drug. However, the path to FDA approval can be a challenge with delays being very common, and there is no guarantee of approval.

When thousands give a little, miracle’s happen. Every share, every dollar, every prayer matters!