Heidi's Hallelujah

03/30/2026

Just a girl cuddling with her doggie before fully waking up.

03/19/2026

https://www.fiercebiotech.com/biotech/rare-disease-advocates-stage-funeral-fda-demand-4-actions-agency-and-congress?fbclid=IwZnRzaAQoKqFleHRuA2FlbQIxMQBzcnRjBmFwcF9pZAo2NjI4NTY4Mzc5AAEeExvK2b6-zk4j97h5HDlO2jvKeaTKjoyJZfbwU9Z3KxB0jJCxFWaZPZ0JQKA_aem_d3_wdCpFiux_FtidLb1kfg

More than 100 marchers took to Capitol Hill on Wednesday after carrying a coffin to the FDA in a symbolic protest highlighting lives they say could be lost to regulatory delays affecting a group of | More than 100 marchers took to Capitol Hill on Wednesday after carrying a coffin to the FDA in a sym...

03/18/2026

This is our reality with Heidi. While we were invited to attend in DC today, we were unable to make it. Every one of these families is faced with the same outcome. We need the FDA to stop denying promising treatments where science has been proven to help provide better quality of life. We are tired of the denials and it’s time the FDA starts actually caring to save our children. Every delay is a death sentence for another child. They are keeping treatment from our families. Even with the treatments that have been constantly denied by the FDA, it actually helps push off the pain and suffering for our kids. It’s been proven effective. We cannot lose the treatments.

03/10/2026

Breaking news - Senator Ron Johnson has "launched an investigation into the Food and Drug Administration's recent denials of treatments for rare diseases."

Sen. Johnson, a champion of early access to treatments for those with serious and life-threatening conditions and author of the Right to Try legislation, again shared his commitment to families during the February 26th Senate Aging Committee Hearing, where Dr. Cara O'Neill, Cure Sanfilippo Foundation's CSO, testified on behalf of patients.

We thank Sen. Johnson and the growing number of Congressional leaders who are increasingly focused on FDA accountability in conducting drug reviews in accordance with congressionally authorized regulatory flexibility.

We want to acknowledge all the Sanfilippo families who courageously shared their stories through the Senate Aging Committee Hearing. We also thank families who have shared the personal impact of delays on their children, particularly since the gene therapy UX111 was initially denied approval in July 2025. This tide of attention to the great unmet need of our children and resulting changes have been supported by months of advocacy from our Sanfilippo community, our MPS advocacy partners, and numerous other rare disease advocacy groups.

There is still much that lies ahead and we will continue to bring the needs of our children forward while working on all fronts with those who can bring about safe and urgently needed treatments.

https://www.newsweek.com/republican-launches-investigation-into-trump-admin-fda-denials-11649790

03/09/2026

Sensory friendly and/or special needs kids, come on out to our church. Heidi will be passing out sensory items and definitely running around to greet everyone.

03/06/2026

While we have to stop driving every 2 hours for Heidi to run, it’s nice to explore new areas and meet new people.

03/01/2026

We need everyone to share this! We need the help of VP JD Vance and President Donald Trump to hear our plea. We would gladly let them meet Heidi and all the other MPS kids if anyone can share this to make it happen. Let everyone in congress know what the FDA is doing and has been doing for years.

02/28/2026

My little rare disease warrior standing tall today for every person fighting a rare disease.

02/27/2026

Heidi is all excited for baseball season!