Your Voice. Their Blueprint.

Patient-Focused Drug Development(EL-PFDD) means a powerful way for families, caregivers, and people living with a disease to directly tell regulatory decision-makers and treatment developers what living with that condition is really like.

EL-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.

This is your chance to shape future treatment options with your voice.

Read on below or download our info sheet here.

The Alexander Disease community will come together for our Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting.

If you’re new to the EL-PFDD, this is a rare and powerful opportunity for our community to speak directly to regulatory decision-makers: sharing the real, lived impact of Alexander Disease and the urgent need for treatments.

This meeting is about US. And only US.

It’s our chance to pull back the curtain and shine a blinding light on what this disease has taken:

  • Children are dying.

  • Brothers and sisters are losing their siblings.

  • Spouses are forced to watch their partners suffer.

  • Parents live with an unimaginable grief no one should ever know.

We live in an age of rapid medical breakthroughs, yet there is still no approved treatment for AxD. If we stay silent, decisions will be made without us.

Our loved ones are not statistics. They are children. They are sisters and brothers. They are partners. They are fiercely loved and they are suffering.

We can’t control every decision, but we can control what we share with those who make them. And that’s where you come in.

We need your voice. We need your story. We need your help.

We Have a Date!

Mark your calendars

December 5, 2025

  • The Goal

    To host an externally-led PFDD (El-PFDD) meeting for Alexander Disease, with regulatory decision-makers attending in a listening role.

    Uncover the real-life impact, unmet needs, and hopes for the future.

    Result in a formal “Voice of the Patient” report that becomes part of the public record.

  • Who should participate?

    Patients living with AxD - newly diagnosed, early onset, adolescent, young adult, adult

    Caregivers and family members of all ages

    Siblings, loved ones, and advocates

    Bereaved families whose voices still deeply matter

    Anyone with lived experience, regardless of diagnosis timeline or disease stage

  • What happens next?

    August 2025:

    Confirmation of participants who have expressed interest will conclude this month

    September - November 2025:

    Kick-off meeting with participants and launch of EL-PFDD survey to community

    Organize panels, survey the community, and prep materials

    December 5th

    We share our stories as one united community

  • How you can help?

    Share your story

    Join our contact registry

    Add your name to the Patient Advocacy support list by emailing: info@endaxd.org

    Help spread the word about the EL-PFDD to fellow AxD families

    Reach out

  • Key takeaways

    This meeting does not involve giving approval or clinical direction - it’s for sharing and listening; for looking into the lives of those affected by AxD

    It’s hosted by the AxD community, not regulators

    Sharing your story increases our chances for the Alexander Disease Community to be heard and to be prioritized for consideration of medical treatments and breakthroughs