Rareatives Feature: A New Chapter for Alexander Disease
A patient-led publication called Rareatives just told the story of this moment in Alexander disease — the first approved treatment, decades of research, and a community determined to be seen. Here’s what it means to us, and where to read the full piece.
The FDA has approved ZANVASTRO™ (zilganersen) as a prescription medicine to treat Alexander disease in children and adults
The FDA has approved ZANVASTRO™ (zilganersen) as the first and only disease-modifying treatment for Alexander disease, in pediatric and adult patients. Here's what the treatment is, what happens next, and where to bring your questions.
A Global Step Forward for Zilganersen
Ionis has partnered with Recordati to bring zilganersen, its investigational Alexander disease treatment, to families outside the U.S. Here's what the agreement means — and what's next ahead of the FDA's September decision.
Ionis Community Update: Zilganersen Program
Ionis shared a community update covering progress across the zilganersen program — including a clinical trial protocol amendment, new AAN 2026 data, and ongoing work to advance global access.
Loudoun Times Feature: The Cancelleri Family’s Story
Loudoun Times Feature: The Cancelleri Family’s Story
Pivotal Zilganersen Study Meets Primary Endpoint; FDA Decision Expected September 22, 2026
The pivotal zilganersen study met its primary endpoint.
FDA decision expected September 22, 2026.
For a community that has lived without any approved disease-modifying treatment, this is a meaningful step forward.
Read the full announcement here.
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…
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