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Patient Registry
Coming Soon!
Alexander Disease is unique, even among leukodystrophies.
It's a very exciting time in leukodystrophy research. Science is progressing rapidly not only for leukodystophies, but especially for Alexander Disease. We are finally able to move past the confusion and emotional devastation of the Diagnostic Odyssey and can look toward a New Frontier: actually bringing treatment to the disorder. As such, we're preparing to build a database of Alexander Disease Patients. This database will quickly prove useful as Ionis Pharmaceuticals looks to bring their drug, ION373, to Clinical Trial as a potential treatment for Alexander Disease.
Having a comprehensive registry as soon as possible not only creates a larger pool of participants for clinical trial, but also gives researchers a more accurate picture of AxD. That means we can potentially identify patients earlier, gain a greater understanding of disease progression, and acheive better patient outcomes.
The Patient Registry will be available soon. Please check back soon and often.
Valentino
Alexander disease is not just a terrible disease; it's a social, financial, and emotional challenge. But our children teach us to deal with it day by day. They are divine beings who came to this life to teach us many lessons. They have a unique angel in their hearts. Valentino can turn the darkest moments into light with just his smile, with the unique love he offers us every day, with his quirks, his sweetness, his intelligence, and intuition. He makes us not give up. He may not understand the meaning of leukodystrophy, but he shows great strength to someday walk, to keep talking, to keep learning.
Isaiah
Today, at almost 5 years old, Isaiah continues to show progress in his motor development. He walks well, eats, and sleeps well and is learning to live without a diaper. He loves watching cartoons with his older brothers Joshua and Matthew. . Just like other 5-year-olds, Chuck E. Cheese is one of his favorite places!
Lilas
We had been waiting 11 years to become parents when we adopted Lilas and her twin brother Silas from an orphanage in Madagascar. They were just 20 months old. There was an obvious difference in the children’s development at first sight. Lilas was held in her nanny’s arms, looking like a 9-month-old baby. She could crawl and stand up holding on to something, while Silas was already walking and running. He had a strong appetite and was beginning to talk. Lilas struggled to eat and could only shout.
Eli
With everything he has gone through and being nonverbal, Eli faces everyday with joy and does his very best to make sure you know what he wants. If that's not God's grace and mercy I'm not sure what is. We are so very proud to be his parents. We pray he can triumph over this disease and do his part in the future of this disease.
Kuno
Kuno is now walking on his own, building up a huge range of vocabularies in both English and Japanese (and a bit of German), talking, singing, and dancing ever so eagerly. He is a kid-model here in Japan. He goes to kindergarten, enjoys regular PTs/OTs as well as swimming. He has powerful genuine smiles which brighten our darkest times, mischievousness, and wit to bring unexpected laughter, and sweet nature to keep us going without falling apart at our weakest moments.
Christopher
We all have the same goal- Cure Alexander’s Disease. We need to come together for this by pooling resource towards research and a cure, advocacy, and awareness. The treatment is amazing and a beautiful opportunity to keep those with AxD as healthy as possible to see that cure. Although Christopher didn’t get to see it, I know he had a part of this amazing path!
Olympia
That was the first time I heard the word Leukodystrophy, that was the day when the pride of all my dreams fell, when I saw the darkness of sorrow, when any sense of future shaded, that was the day I faced love, the bare form of it, unveiled from all the superficiality of built expectations. That day I became the true mother of Olympia. That day I painfully understood what it meant to be a mother.
Hailey
Hailey was six years old when she got the diagnosis. She continued to attend school, with the help of an aid ,and this May at age 20 graduated from high school. Hailey loves to draw in her sketchbook, enjoys watching cooking shows, and wants to be a chef when she grows up. She has quite a collection of Barbie dolls, loves the color pink, shopping , and trying new restaurants.
Aidan
July AxD Featured Fighter
Aidan is very aware of his diagnosis and chooses to fight the disease and its daily manifestations. None of this deters his spirit. He does not define himself by this disease, he says it is nothing more than another trait.
Chayse
June Featured AxD Warrior
Chayse is the youngest of three boys and ironically the largest at birth however, it wasn’t long till he showed signs of being a pickier eater than his other two brothers had been. He would never really eat normal baby food and took his bottle for longer than I had ever let the other two. We didn’t think too much of it and chalked it up to just pickiness.
Megan
May Alexander Disease Featured Angel
Our daughter Megan’s story is similar to the other families in the AxD community. She went through years and years of countless tests to gather an idea of what was happening in her tiny body. Her initial symptoms were slow to present and seemingly without connection with each other, so her story is long and varied. Unfortunately, the ending to our story is different than the other featured AxD warriors, although it’s no less impactful.
Daniel
April Featured Alexander Disease Warrior
Daniel Hughes was born April 19, 2010. At 2 ½ years old, we were told to take Daniel home and just give him the best life possible and there was nothing we could do but treat symptoms and keep him comfortable – that he wouldn’t live past his 6th birthday. After the initial shock, we refused to accept there was nothing we could do.
David
March Featured Alexander Disease Patient
David was born a healthy and happy baby. He wasn’t a great sleeper and spit up all the time, but babies do that, right? Some of his early milestones were also a little slow in happening but babies develop at their own pace, right? When he didn’t get up on all fours to crawl but rather chose to get around on his belly, I thought he was adorably quirky.
Anna
February Featured Alexander Disease Patient
Anna Walentowski will turn 23 in February.
Our journey with Anna’s Alexander Disease diagnosis began over 21 years ago .During infancy Anna had some feeding difficulties, was slow to achieve gross motor milestones and her head size increased rapidly between 4 and 12 months while her weight started dropping off her curve. Her pediatrician recommended a brain CT scan at 15 months, then MRI at 18 months of age. The results revealed “an abnormality of the white matter consistent with leukodystrophy”.
Mila
January featured Alexander Disease Patient
Mila was born a healthy baby. Nahed and Gil Sarig were thrilled as they had experienced several miscarriages while trying for a second child. Mila’s arrival was truly a blessing.
Jordyn
December Featured Alexander Disease Patient
In the spring of 2015, Jordyn Conn wasn’t walking yet. She had turned one, the previous December, but her parents were not overly concerned. In March, Jordyn began having tremors in her upper body which caused her to lose her balance when she crawled sometimes hitting her face on the floor. She also lost the ability to turn pages in her board books.
Meris
November Featured Alexander Disease Patient
At 9 months old, Meris White was gagging and retching when her parents attempted to feed her pureed baby foods. Still, at her 9 month doctor visit, pediatricians didn't seem concerned. Meris' parents, Qwynn and Brandon White, weren't convinced that this was a typical delay, so they continued to press for answers. While many first time parents are dismissed as being overly cautious, the White's decided not to ignore their instincts and reached out to the Tennessee Early Intervention System (TEIS) on their own to have Meris evaluated.
Elise
Born in September 2011, Elise Bonsky is a bright, vibrant 8 year old. Early in life, she suffered from a few developmental delays and seizures, but while the Bonsky family made sure she didn't outgrow her dosage of Keppra and had annual MRI's, they had little cause to suspect she might have a devastating underlying condition. Then, in November of 2014, the Bonsky family received a phone call from her local neurologist that changed everything. The tone of voice said it all: Something in Elise's MRI had changed and they needed to get to the hospital as quickly as possible.
Cameron
Cameron’s family has a motto: "Can't Never Could." Cameron Copeland is a twenty-three year old young man who grew up doing everything from riding bikes, to participating in Culinary Programs, to being on his school Archery Team for 4 years.
Grayson
Grayson’s story began well before he was born. At 20 weeks old in the womb, complications threatened his life; his parents, Josh and Laura, prayed and hoped for a chance to get to know him. He was born the following summer to beaming parents.
Now, as an eight-year-old living with a seemingly insurmountable disease, we have hope. Hope for a treatment and eventual cure. With research and funding, this treatment could be the difference for people like Grayson, and we are determined to be a catalyst for that cause.