Asa’s Army Against Alexander Disease

02/02/2026

💙🦓🧬

Did You Know?
Alexander disease is an ultra-rare genetic disorder,
affecting about 1 in 1 million people worldwide.

01/30/2026

REMINDER: We need your stories!

Stories help people understand what living with rare disease truly looks like.
❤️ They inform advocacy efforts.
❤️ They help guide research priorities.
❤️They remind families they are not alone.

If you have been considering sharing your story and a lovely picture, we encourage you to do so.

https://forms.gle/kCq1iPWN8xjJjzKA8
Important note: Because this form includes photo submissions, respondents will be asked to sign in using an email account that is tied to a Google account in order to complete the form. This is a requirement set by the form platform to allow photo uploads. Your information is not shared beyond this submission. You are not required to submit a photo, but this form does require at the minimum of a story.

If you are unable to access the form but would still like to participate by sharing your story and emailing a photo, please reach out to us at info@endaxd.org and we will work with you.

Please request to receive your story and photo by Friday, February 6th.

Every voice adds meaning and strength to this community.

01/21/2026

💙🦓

Every story in this community matters.

As we head into a new year, we are inviting individuals and families impacted by Alexander Disease to share their story and snapshots with us. These stories and submitted images will help raise awareness, strengthen advocacy efforts, and remind families they are not alone.

https://forms.gle/kCq1iPWN8xjJjzKA8

Important note: Because this form includes photo submissions, respondents will be asked to sign in using an email account that is tied to a Google account in order to complete the form. This is a requirement set by the form platform to allow photo uploads. Your information is not shared beyond this submission. You are not required to submit a photo, but this form does require at the minimum of a story.

If you are unable to access the form but would still like to participate by sharing your story and emailing a photo, please reach out to us at info@endaxd.org and we will work with you.

Please request to receive your story and photo by Friday, February 6th.

Your voice matters, and we are grateful for your willingness to share.

01/12/2026

Great news for our Alexander Disease community💙🧬

Ionis Pharmaceuticals has announced the opening of an Expanded Access Program (EAP) in the United States for individuals with Alexander disease, which may allow eligible patients to seek access to the investigational therapy zilganersen outside of a clinical trial.

Expanded access, also referred to as compassionate use, is one potential pathway for people with life-threatening diseases to gain early access to an investigational medicine when no satisfactory alternative treatment is available.

We’re sharing this information so our community is aware of available pathways and can have informed conversations with their medical teams.

For full details and to learn more, visit: https://ionis.com/patients-and-advocacy/expanded-access-policy

01/12/2026

💙💙

Anticipatory Grief

This isn’t something I particularly like to speak about for obvious reasons.

But when my feelings get the best of me I cope by writing and sharing those feelings.

So here we are.

When my child was diagnosed with a life limiting condition I was struck very quickly with a sense of grief.

Before I could even fully process what doctors had told me, a million thoughts had run through my mind “I’m never going to hear mama” “I’m never going to see her do this or that” “I’m never going to experience normal again”.

This grief hurt, but this grief was expected.

I had lost the child I thought I was going to have.

Over time that grieve faded.

We fell into our own sense of normal.

But with her prognosis also came anticipatory grief.

A feeling of inevitable loss.

Waiting for that moment where everything falls apart.

It isn’t always at the forefront, but it is always slightly lingering.

With every seizure,

with every illness,

with every hard day and hard decision, that lingering feeling grows.

It’s a hard truth that cannot be escaped.

Nobody prepares you for this feeling of grief.

Nobody warned me I was going to grieve my child so deeply while she’s still living.

Nobody explains that it is a normal feeling for parents of children with a life limiting illness.

So, I’m here to say anticipatory grief is real.

It’s raw.

It’s emotional,

AND it is normal.

Be kind to us moms when we speak about losing our child who is still here.

We are not morbid,

we are not pessimistic,

we’re simply living out a reality that most are lucky enough to not understand.

Written by: Carla Moore from Payton's Path

12/25/2025

12/12/2025

A step in the right direction💙💙

A meaningful milestone for the Alexander Disease community.

Ionis has received U.S. FDA Breakthrough Therapy Designation for zilganersen for AxD, an important recognition designed to accelerate the development and review of therapies with the potential to offer substantial improvement for serious or life-threatening conditions.

This designation does not mean approval, but it does mean momentum.

Read the full announcement here:
🔗 https://ir.ionis.com/news-releases/news-release-details/ionis-receives-us-fda-breakthrough-therapy-designation

End AxD will continue accelerating research, strengthening advocacy, and ensuring the AxD community remains at the heart of every advancement.

12/12/2025

💙🧬

You heard the stories -the challenges, the resilience, the urgency.

Now, we take action.

Your gift helps us continue the work that matters most:
• accelerating research
• supporting families
• advocating for real change in Alexander Disease care and treatment

If the PFDD conversations moved you, inspired you, or opened your eyes to the reality of living with AxD then it's clear that your donation today fuels the future we’re fighting for.

👉 Give today: endaxd.org/donate

Every contribution, large or small, builds the foundation for what comes next.

11/25/2025

As a proud partner of End Alexander Disease ( End AxD ), the United Leukodystrophy Foundation invites our community to rally around the Alexander Disease community during their upcoming EL-PFDD meeting on December 5.

This meeting gives families and patients affected by Alexander Disease the chance to share their experiences directly with those shaping future research and treatments.
You can show your support by joining virtually. Whether you tune in for the whole session, or just part of it - your presence matters.

December 5 | EL-PFDD Virtual Event

🔗 Register here: www.endaxd.org/endaxdel-pfdd

Together, we can amplify the voices of the Alexander Disease community and drive awareness for all impacted by leukodystrophies.

11/25/2025

In this season we are extra thankful for this little guy & all the hard work he puts in daily!! 💙💙 Asa has more appts coming up in December along with some exciting things moving forward to hopefully get a treatment on the market for Alexander disease in the near future..prayers for these appts and meetings go well are much appreciated 🥰🧬