Saving Sadie Rae

Commissioner Makary, You fast-tracked weight-loss drugs in <55 days. My child has Sanfilippo, a type of childhood dementia. There are no approved treatments. UX111 has 10+ years of data. Kids on it are thriving while mine is dying. Please save our children.

@MarshaBlackburn @BillHagertyTN @ThomTillis @TedBuddNC

This week is critical. We will be in DC advocating for change. - Today, Dr. Cara O’Neill is speaking at a joint FDA/Duke Margolis event about controls in rare disease clinical trials. - On Thursday, we will attend a Senate meeting where Secretary Kennedy will testify….

@VPrasadMDMPH @US_FDA @MartyMakary @DrMakaryFDA @SecKennedy #ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary

We are urging our elected officials to delve deeper into the issues surrounding rare disease delays at the FDA. - From Wednesday to Friday, we will be meeting with various Congressional offices and White House staffers.

thehill.com/opinion/health… @MartyMakary @VPrasadMDMPH @US_FDA @SecKennedy @SenatorWicker @RonJohnsonWI @RogerMarshallMD @BillCassidy @RepGusBilirakis @amyklobuchar @DorisMatsui

Behind every delay is a child losing skills, a family watching helplessly. These aren’t statistics. They’re kids. And they don’t have time to wait. Link to full article in comments. 📩 Take action at ApproveHopeNow.com #ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary

The science is ready. The system is not. Kids with ultra-rare diseases like Sanfilippo are losing abilities because the FDA uses outdated processes built for large populations. Time is brain. If the science is solid, get treatments to patients NOW. biocentury.com/article/656694…

Today’s the last day. The FDA delayed a gene therapy for Sanfilippo — a disease that steals children’s voices, mobility, and memories. We can’t wait another year. 📣 Sign the letter before midnight: tinyurl.com/UNC-RareDis #ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary

Tell the FDA: kids with Sanfilippo can’t wait. A promising gene therapy was delayed up to a year over paperwork. Sign the letter before Aug 6 to demand urgency. 🔗 unc.az1.qualtrics.com/jfe/form/SV_0i… #ApproveHopeNow #RareDisease #Sanfilippo #NoMoreDelays

We know @MartyMakary & @VPrasadMDMPH care about rare disease families. Please act on your words. Meet with us. Help review this quickly once resubmitted. Our kids are losing their voices. We need yours. #ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary

The @US_FDA said the data behind this Sanfilippo gene therapy is strong. But instead of resolving minor manufacturing issues, they rejected the application. Now families must wait 6–12 more months. In Sanfilippo, that’s a lifetime - speech lost, mobility gone, memories erased.

🇺🇸 Happy 4th y’all! 🇺🇸

We’re looking for auction items and sponsors for 🦋Sadiepalooza🦋. On August 24 and 25, we’ll be hosting a 5k, golf tournament, silent auction, and more in Troy. If you or your company would like to contribute to the silent auction or sponsor one of the events, please let us know

@katyperry Thank you! Our sweet 2 year old, Sadie, has the same terrible disease. #savingsadierae #savingcarter #curesanfilippo

My ❤ was touched by Carter's story. If you're able pls help w/ his chance to defeat Childhood Alzheimer's. He's SO close to funding his clinical trial & I would love to see my KatyCats kick it over the goal & make that difference! I’m donating now too! 🤗 gofundme.com/savingcarter

@KrisJenner Thank you! Our sweet 2 year old, Sadie, has the same terrible disease. #savingsadierae #savingcarter #curesanfilippo

🙏 Please help save little Carter's life!! Carter has a version of childhood Alzheimer’s called Sanfilippo Syndrome and his family need to raise awareness and funds to get Carter the clinical trial he needs to save his life!! Every little helps. 🙏 gofundme.com/savingcarter

Sadie loves a good selfie! I’m still not sure how she does the photo effects! #nationalselfieday #selfie #selfieday

@VancityReynolds Carter is 6 and suffers from a childhood Alzheimer’s. Our 2 year old daughter, Sadie, has the same terrible disease. We have 3 days left to reach the goal to give them both a chance at life! Please watch, share and donate! SavingCarter.com #SavingCarter

@blakelively Carter is 6 and suffers from a childhood Alzheimer’s. Our 2 year old daughter, Sadie, has the same terrible disease. We have 3 days left to reach the goal to give them both a chance at life! Please watch, share and donate! SavingCarter.com #SavingCarter