Global Alexander Disease Community Unites in Open Letter to Ionis
On February 9, 2026, End AxD joined leading international Alexander disease and leukodystrophy organizations in submitting a unified open letter to Ionis Pharmaceuticals in response to the opening of the U.S. Expanded Access Program for zilganersen.
This letter, signed by advocacy leaders across North America, Europe, Australia, Japan, and beyond, reflects a powerful and coordinated global voice. Together, we expressed both deep gratitude and urgent advocacy.
We are sincerely thankful for the opening of the Expanded Access Program in the United States. For families facing serious and life threatening progression, access to investigational treatment outside of clinical trials represents more than hope. It represents possibility.
At the same time, we stand united in calling for urgency for international families. The development of zilganersen has been made possible by patients and clinical sites across multiple countries. We believe access should reflect that global commitment.
The letter respectfully urges Ionis to accelerate the implementation of Expanded Access Programs internationally, recognizing the extreme urgency facing families outside the United States.
This milestone follows years of advocacy, including the Externally Led Patient Focused Drug Development meeting where our community made clear the enormous unmet need. Our collective message is simple. No patient should be left behind because of geography.
We are proud to stand alongside:
• The United Leukodystrophy Foundation
• Alex TLC
• ELA International
• The Italian Alexander Disease Association
• The Spanish Association of Alexander Disease Patients
• Leukodystrophy Australia
• Additional global representatives and families
This letter reflects what is possible when a rare disease community moves together with clarity, collaboration, and shared purpose.
We remain committed to accelerating research, advocating for access, and ensuring that every family affected by Alexander disease is seen, heard, and supported.
You can read the fully signed letter here.
