Your Voice. Their Blueprint.
Voice of the Patient Report
On December 5, 2025, the Alexander Disease community came together for an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting to tell regulators, researchers, and drug developers what living with AxD really looks like.
The Voice of the Patient Report is the formal output of that meeting — a synthesized record of what the community shared: the day-to-day realities of life with Alexander Disease, how families approach managing it, and the priorities and unmet needs that should shape future treatments. The Report becomes part of the official documentation that the FDA, drug developers, and researchers turn to when making decisions about AxD.
The three documents below preserve that record in full — the Report itself, along with the written comments submitted by patients, caregivers, and family members around the world, and the poll responses from participants at the meeting.
Watch the EL-PFDD recording from December 2025
Patient-Focused Drug Development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs and priorities are captured and meaningfully incorporated into drug development and evaluation.
Externally - Led PFDD meetings (EL-PFDD) are one such approach. These meetings are a powerful way for patients, their families, caregivers and patient advocates impacted by a disease to directly tell regulatory decision-makers, treatment developers, academic researchers, and healthcare professionals what living what that condition is really like and what meaningful treatment look likes from the patient and caregiver perspective.
Learn more via the download info sheet here.
On December 5th 2025, the Alexander Disease community came together for a virtual Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting, hosted by End AxD.
It was a rare opportunity for our community to speak directly to regulatory decision-makers, treatment developers, researchers, and healthcare professionals — sharing the real, lived experience of Alexander Disease and the urgent need for treatments.
This meeting was about US. And only US.
Ahead of the meeting, a pre-meeting survey gathered input from families, caregivers, and people living with AxD around the world. Those responses helped shape the conversations on December 5 and informed the questions stakeholders brought into the room.
What the community shared:
The day-to-day effects of Alexander Disease, and how they change over time
How families approach managing and treating AxD
Priorities and preferences for new treatment options
What the meeting accomplished:
A clearer picture of unmet needs in current AxD treatment approaches
A formal record researchers and regulators can return to as they make decisions about Alexander Disease
All of this is now captured in the Voice of the Patient Report, with the supporting documents available at the top of this page.