Saving Sadie Rae

Sweet Sadie has a fatal disease called Sanfilippo Syndrome (MPS IIIA). Please join us on our journey to raise awareness and funding to cure Sadie.

[Saving Sadie Rae @SavingSadieRae]

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.

[Saving Sadie Rae @SavingSadieRae]

@MarshaBlackburn @BillHagertyTN @ThomTillis @TedBuddNC

[Saving Sadie Rae @SavingSadieRae]

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….

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

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.

[Saving Sadie Rae @SavingSadieRae]

https://t.co/K04FLsmzOb

@MartyMakary @VPrasadMDMPH @US_FDA @SecKennedy @SenatorWicker @RonJohnsonWI @RogerMarshallMD @BillCassidy @RepGusBilirakis @amyklobuchar @DorisMatsui

[Saving Sadie Rae @SavingSadieRae]

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 https://t.co/Kw2ziE23kT

#ApproveHopeNow
#NoMoreDelays #WalkTheTalkMakary

[Saving Sadie Rae @SavingSadieRae]

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Kids with ultra-rare diseases like Sanfilippo are losing abilities because the FDA uses outdated processes built for large populations.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.

https://t.co/lbhVVwr7M6

[Saving Sadie Rae @SavingSadieRae]

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: https://t.co/Chn2iIRA7h

#ApproveHopeNow #NoMoreDelays #WalkTheTalkMakary

[Saving Sadie Rae @SavingSadieRae]

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.
🔗 https://t.co/iGvFiza3P1
#ApproveHopeNow #RareDisease #Sanfilippo #NoMoreDelays

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

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.

[Saving Sadie Rae @SavingSadieRae]

🇺🇸 Happy 4th y’all! 🇺🇸

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

@katyperry

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

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

[Saving Sadie Rae @SavingSadieRae]

@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!

https://t.co/ZSliJ0xeNV
#SavingCarter

[Saving Sadie Rae @SavingSadieRae]

@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!

https://t.co/ZSliJ0xeNV
#SavingCarter