Announcing the 2025 End AxD Grant Program Recipients
End AxD is proud to announce the recipients of the inaugural 2025 End AxD Grant Program. Following a review of 24 applications from around the world, the End AxD Scientific Advisory Committee has recommended five pioneering research projects for funding.
This year’s program represents an initial 2025 commitment of $263,419 with potential for year two investment of $266,928, pending mid-term progress reports.
Global Alexander Disease Community Unites in Open Letter to Ionis
The global Alexander Disease community has spoken with one voice. In a unified open letter to Ionis Pharmaceuticals, advocacy leaders and families expressed gratitude for U.S. expanded access to Zilganersen while calling for accelerated international implementation to ensure equitable access for patients worldwide.
Children’s Hospital of Philadelphia Names Dr. Amy Waldman Endowed Chair in Alexander Disease Research
Children’s Hospital of Philadelphia Names Dr. Amy Waldman Endowed Chair in Alexander Disease Research
Dr. Dolores Pérez-Sala’s team - Research Update
Dr. Dolores Pérez-Sala’s team - Research Update
Dr. Yukari Blumenthal - UMass Research Update: A "Mute Button" for Alexander Disease?
Dr. Yukari Blumenthal - UMass Research Update: A "Mute Button" for Alexander Disease?
Dr. Ylenia Vaia - 3 Big Clues They’re Investigating
Dr. Ylenia Vaia - 3 Big Clues They’re Investigating
Dr Hagemann is Investigating Sickness and Eating Issues
Dr Hagemann is Investigating Sickness and Eating Issues
A Mother’s Promise, A Community’s Mission
End AxD board member Deborah Hughes turned the pain of her son Daniel’s Alexander Disease diagnosis into purpose. Through advocacy, fundraising, and research support, she carries his light forward—driving hope, progress, and a future with treatments for Alexander Disease.
Pranam Chatterjee, recipient of EndAxD funds to study AxD, receives NIH grant to expand AI platform to target diseases
"The EndAxD award was instrumental in getting the NIH grant and the projects are very complementary. The results of the R35 will help us be more specific in targeting GFAP and I look forward to continuing to work on Alexander Disease with our research."
- Pranam Chatterjee
IONIS Update on Clinical Trial
Read the letter below for more information and details from IONIS…
Astellas Announces Sponsored Research Agreement with UMass Chan Medical School
Astellas Pharma US, Inc. has teamed up with UMass Chan Medical School to advance research on a groundbreaking gene therapy for Alexander disease, an ultra-rare and fatal condition…
Thank you …
Just wanted to say THANK YOU for making Golf Fore Grayson 2024 a huge success!
Empowering Families Affected by Alexander Disease Through Groundbreaking Research
We are excited to share that End AxD has funded the innovative research conducted by the Chatterjee Lab at Duke University…
End Of Year Giving Invitation
As we celebrate the holidays, we are grateful for the blessings of community. 2023 has been a tough year for so many, but we remain grateful and hopeful.
Save the Date! Golf Fore Grayson! - April 29th, 2024
We are back on the calendar for the 3nd annual Golf Fore Grayson!
United Leukodystrophy Foundation Conference '23 - AxD Session Links
We're excited to share some highlights from the recent United Leukodystrophy Foundation conference, where the latest advancements in Alexander disease research and treatments were shared. Here's a recap of the sessions you won't want to miss:
Webinar Recording Available Now: AAV-medicated Gene Silencing (August 2023)
If you weren't able to join us on Sunday, August 6, 2023 for Dr. Gao’s update regarding “AAV-mediated Gene Silencing for the Treatment of Alexander Disease”
End AxD funds gene therapy research for Alexander disease
The foundation End AxD, a nonprofit dedicated to research, treatment and a cure for Alexander disease, has donated $74,000 to UMass Chan Medical School to explore a gene therapy treatment for the fatal neurological disease.