10/29/2025
Please share and get the word out on this incredible opportunity, and if you are able to please consider donating!
BIG ANNOUNCEMENT!
The Sanfilippo community has been presented with an opportunity to have an Expanded Access enzyme replacement treatment for some children with Sanfilippo Type B.
As of the past couple of weeks, an opportunity with a company to create drug product (medicine) for children with Sanfilippo Type B has been brought forward.
There are many details that remain unknown regarding the exact path forward, including the criteria for children to be part of the Expanded Access, the treatment site locations, the exact timing, how children are selected, and much more. Appropriately, Cure Sanfilippo Foundation will not be a part of choosing who would be accepted into this Expanded Access. Eligibility and enrollment would be determined by the doctors.
We have been told this could supply up to 14 children with one year of enzyme replacement therapy treatment. The path beyond that time is uncertain. There is hope that the company’s therapy would become FDA approved during this time and patients receiving Expanded Access would then be able to receive the approved drug. As we know, however, the path to FDA approval can be a challenge and delays are common, and there is no guarantee of approval.
So what do we know? We know there is a drug manufacturing slot available to create this Expanded Access drug. This slot has to be secured by Dec. 1, and the costs for this are falling on the patient community. This is not the typical way for Expanded Access to be funded; however, given the extreme unmet need, we look for any opportunity to increase access to treatment.
The cost by Thanksgiving this year (1 month away) is about $4 million dollars. Beyond that, there will be additional estimated costs of about $2 million to be raised in 1Q 2026. Cure Sanfilippo Foundation and our partners at the National MPS Society, in coordination with Sanfilippo Type B families, have fundraisers active for this project.
So we are putting a call out there to all supporters of Sanfilippo Type B children: Help us raise as much as possible in this short window of time, and thank you for considering.
We expect to hear additional information soon from the company on the details and plan.
For now, it is full-on fundraising! Here’s how you can help:
💲 Donate online; links to online campaigns below
☑️ Donate via check, ACH, or other ways available to give.
📫Most importantly, please share these ways to contribute. On social, in email, everywhere you can.
- Donate via the Kempf family page, and watch their inspiring video about both of their children who were diagnosed with Sanfilippo Type B: https://give.curesanfilippofoundation.org/campaign/732486/donate
- Donate via the Cure Sanfilippo general donation page: https://give.curesanfilippofoundation.org/campaign/653569/donate In comment field type: "Expanded Access Type B"
- Donate via check, written out to sent to: Cure Sanfilippo Foundation, PO Box 6901, Columbia SC 29260. On memo line, write "Expanded Access Type B"
***In the event that this program is not able to move forward, the directed funding raised for this initiative will be applied to alternative Sanfilippo Type B treatment pathways***
