Elise's Corner

09/22/2025

**warning: Long and emotional post**

To the entire crew at Ionis Pharmaceuticals, I want to take a moment to thank you for your unwavering support. When I first stepped (back) into the advocacy world, I feared that our community would be seen as numbers on a page rather than as people in need.

But Ionis has proven me wrong in the most beautiful way. Your compassion, your commitment, and your humanity shine through everything you do. To me, you’re not just a team—you’re family, standing alongside me, my sweet Elise, and so many others across this community.

It is an honor to walk this journey with you. I can only imagine the celebrations inside Ionis today—the high fives, the smiles, the tears of joy (shh I won’t tell) —as you thought about who this treatment will touch. Knowing that you share in those emotions makes all the difference.

For nearly 11 years, my prayers have been the same: that no mother would ever again walk into a doctor’s office with her beautiful three-year-old little girl and be told, “there is no treatment.” I have lived that moment. I have seen how a single sentence can shatter a mother’s very existence—how the world turns upside down, how the future disappears, how hope feels stolen in an instant. No parent should ever carry that kind of pain. And today, for the first time, I believe those prayers are so close to being answered.

My favorite thing Elise says – without fail, every single night – and without any prompting – is, “I love you, Momma.” Those four words are my treasure and make me need to carry her fight forward. And yet, every night, my heart breaks a little, knowing this disease will one day try to silence those beautiful words. I know there will come a night when it will be the last time I hear those words in her sweet voice. That thought has haunted me for years. But, with this news, I finally have hope that maybe – just maybe – that night will never come.

I owe you all so much for helping us get so incredibly far.

Today, my world feels brighter, my heart feels more whole, and I believe I may have taken my first real breath in nearly 11 years. For choosing not only to invest in this rare disease trial, but to pour your hearts and your science into it—I owe Ionis more than words can ever express.

I’ve often shared my “ugly snow globe” metaphor. Well, today, that snow globe just became a whole lot more beautiful—because of Ionis.

Cheers to the success we’ve realized thus far…and may we use this positive momentum to driving even greater impacts forward so that no beautiful child ever has to be given a death sentence before they get a chance to live.

We are deeply, profoundly grateful and love you all beyond words.

09/22/2025

Today is the day so many of us have dreamed of and prayed for. From now on, no mother will sit in a hospital or doctor’s office and be told there is no treatment for Alexander disease. That precious three-year-old girl now has hope—hope for a therapy that can change the course of her life. And her parents will have the gift of watching her grow.

𝗦𝘁𝗮𝘁𝗲𝗺𝗲𝗻𝘁 𝗳𝗿𝗼𝗺 𝘁𝗵𝗲 𝗘𝗻𝗱 𝗔𝘅𝗗 𝗕𝗼𝗮𝗿𝗱 𝗼𝗳 𝗗𝗶𝗿𝗲𝗰𝘁𝗼𝗿𝘀

The Alexander Disease community is stronger and more united than ever. Advocacy has opened doors, research has accelerated, and the world is paying attention.

This marks a milestone, but it is just one step in a much larger movement forward. For the first time, a therapy has demonstrated a clinically meaningful impact in Alexander Disease, a monumental step forward for a rare condition that has long had no treatment options.

As Ionis shared in today’s announcement, “𝘛𝘰𝘥𝘢𝘺’𝘴 𝘯𝘦𝘸𝘴 𝘪𝘴 𝘢 𝘮𝘰𝘯𝘶𝘮𝘦𝘯𝘵𝘢𝘭 𝘴𝘵𝘦𝘱 𝘧𝘰𝘳𝘸𝘢𝘳𝘥 𝘪𝘯 𝘢𝘥𝘷𝘢𝘯𝘤𝘪𝘯𝘨 𝘢 𝘱𝘰𝘵𝘦𝘯𝘵𝘪𝘢𝘭 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘧𝘰𝘳 𝘈𝘭𝘦𝘹𝘢𝘯𝘥𝘦𝘳 𝘥𝘪𝘴𝘦𝘢𝘴𝘦, 𝘰𝘧𝘧𝘦𝘳𝘪𝘯𝘨 𝘭𝘰𝘯𝘨-𝘢𝘸𝘢𝘪𝘵𝘦𝘥 𝘩𝘰𝘱𝘦 𝘧𝘰𝘳 𝘱𝘦𝘰𝘱𝘭𝘦 𝘭𝘪𝘷𝘪𝘯𝘨 𝘸𝘪𝘵𝘩 𝘵𝘩𝘪𝘴 𝘤𝘰𝘯𝘥𝘪𝘵𝘪𝘰𝘯, 𝘵𝘩𝘦𝘪𝘳 𝘧𝘢𝘮𝘪𝘭𝘪𝘦𝘴 𝘢𝘯𝘥 𝘵𝘩𝘦 𝘤𝘰𝘮𝘮𝘶𝘯𝘪𝘵𝘺.” This echoes what our families, advocates and researchers have worked toward for years - the day Alexander Disease could no longer be ignored.

With the EL-PFDD meeting ahead, we have another historic opportunity to make our voices heard and to shape the future of treatments and care.

Hope is alive, momentum is real, and together we will continue driving toward lasting change.

To learn more about the study results announced today, you can read the full Ionis news release: https://ir.ionis.com/news-releases/news-release-details/ionis-announces-positive-topline-results-pivotal-study

Please ensure you are part of our contact registry to stay informed: https://www.endaxd.org/patients-and-families

09/13/2025

This past week has been so heavy for our country, yet in the midst of that darkness, Elise and I shared one of the most profoundly beautiful human experiences yesterday. We will share more later, however, we’ll leave this picture here as a “placeholder” till we have more time. 😉

In the meantime, sending so much love, hope, and prayers for everyone, everywhere.

09/02/2025

It’s a rare and devastating leukodystrophy that affects the brain and spinal cord. Caused by a mutation in the GFAP gene, it leads to the buildup of toxic proteins that damage myelin — the protective coating around nerves.

There is currently no approved treatment. But there is hope.

Through research, advocacy, and the strength of our community, we believe the future of Alexander Disease can change.

👉 This September, during Leukodystrophy Awareness Month, follow along with End AxD as we spotlight more about Alexander Disease, share the voices of our community, and highlight the work being done to bring answers and hope.

🔗 Learn more: www.endaxd.org

09/01/2025

Happy 14th birthday to the most magically beautiful, strong and innocent girl.

We don’t post here often these days because, well, life is rough. It’s so crazy seeing all of Elise’s sweet friends grow older while she continues to take a different path. We celebrate all of her milestones and love every single day we get to spend with her.

She’s transformed my life in ways I never knew possible. The love that her sister has for her is something that can’t be taught. Emmersyn Is the most natural caretaker of everyone around her, but especially “her” Elise. Ems and I are blessed beyond words to walk this journey alongside our Elise.

Birthdays are always emotional for mothers. They’re such a beautiful reminder of the days in which we met the children we call our own. The days that we looked at their sweet faces and had all the hopes and dreams for them.

But birthdays are also so hard, especially for moms with children with life limiting diseases. While we honor how much strength and fighting has gotten us here, it also feels like a countdown to something unimaginable.

None of us know when our clocks will come to an end, but it feels especially selfish when a beautiful child has one ticking down. It’s the worst kind of unfair there is.

While this life is hard, I’m always reminded that no one ever promised that life would be easy.

Today, we’re celebrating the animal-loving, Taylor Swift obsessed, peanut butter monster that always finds my mom jokes funnier than necessary. While we celebrate her, I also cant help but just fee a little sad.

Elise is our entire world. Ems and I orbit around this sweet girl in life and we’re so very proud to call her our very own. Praying that we get so, so many birthdays with sweet E.

We love you, Leesie girl 💗💗💗