Did You Know

09/29/2026

Fun App for improving various mental skills. Who’s game?! 😊 ...

09/25/2026

It's International Ataxia Awareness Day. Tell me below what you're doing today.

09/24/2026

September 25th remember Ataxia awareness, join me in telling your state rep about Ataxia

Published on Oct 15, 2017Dana Mauro, The National Ataxia Foundatio...

06/22/2026

Episodic ataxia is a group of related conditions that affect the nervous system and cause problems with movement

iTunes: podcast/k...https://itunes.apple.c...

https://youtu.be/vr51CgnOZQU?feature=shared

Episodic ataxia is a group of related conditions that affect the ne...

05/29/2026

Your next doctor visit I have be prepared easy tips.

Today’s topic is Doctor VisitsSo how many of you can relate to thi...

04/17/2026

04/13/2026

Love looking back and remembering past this is 8years ago

Thank you for supporting research so many great events. What an amazing effort by so many! SUBSCRIBE to our Channel and share if you agree!

04/11/2026

The extreme hike for was the most rewarding experience I have had

04/11/2026

Extreme hiking for was most rewarding thing I have ever done.

03/12/2026

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