11/02/2025
Some of you may recognize our friend Liv. She was diagnosed just a week after JoJo and has Sanfilippo Type B. As JoJo and I are headed to the airport for treatment through a clinical trial it only seems right I share this opportunity to give type B kiddos the same chance. Liv's mama has written it out so well as it is a unique situation and I know times are hard, the holidays are coming, times are extra extra hard at the moment but even if you cant give but only take a minute to share it can make a big difference. It's crazy there is a treatment that shows it works and yet these families don't have access to it. Help save these sweet babies.
Saving Liv
Update: We are at 1 million collectively for all families raising money!! We still need 2.8 more 🙏
BIG ANNOUNCEMENT!
There is an opportunity for “early access” or “compassionate use” for an enzyme replacement medication for kids with mucopolysaccharidos type 3B (Sanfilippo Syndrome Type B ). Liv’s type!
Liv was diagnosed in March of 2022 when she was 2, and she’s now almost 6 (on 12/3) and there have been zero medication options available. Supportive measures only.
Liv is lacking an intracellular enzyme that breaks down cellular waste. Since this waste product is found in all cells, it affects every organ, including her brain. She’s already lost her speech, and will lose her ability to feed herself and walk in the coming years. She’s cognitively regressed to about a 1 year old.
For the first time ever for Sanfilippo Syndrome, a biotech company will be making extra medication for compassionate use. This is an enzyme replacement therapy, a weekly infusion directly into an Ommaya reservoir in the brain. There was a trial that ran out of money years ago, that showed great promise. This new biotech has purchased the rights to the drug and all the data from the trial. This company will also be applying for FDA approval in 2026. They are extremely confident in the data and are very hopeful for approval, but nothing with the FDA has come quickly.
But we have seen how quickly this disease takes over. Liv and her Sanfilippo friends don’t have time to wait for the FDA to approve this.
The biotech company is fundraising to make more life altering medication for kids like Liv. They are asking for $6 million dollars by February (with over half by Thanksgiving). To me that number is insane. Out of reach, unattainable. This would be for 14 kids to get the infusion for a year. But I have to try, not only for Liv, but for the sake of progress and newly diagnosed kids who haven’t yet had significant brain damage.
Whether or not Liv can get it, or would be appropriate for it when it’s available, is yet to be determined. But this is extremely promising and hopeful for newly diagnosed families.
If you have it in your hearts and wallets, I am shamelessly asking for a small donation to our GoFundMe SavingLiv . Com - this all goes directly to the Cure Sanfilippo Foundation. I’ll add the link in the comments.
If you’ve made it this far, all my gratitude and appreciation 💜
