My daughter with FA dreams about reversing her disease progression

I am Amelia's paternal grandfather. I held her in my arms in the hospital the day she was born. We had a long conversation. Mostly unspoken. But a profound connection. None of us understood just what she was going thru until she was diagnosed. But she was not thriving. Then she was put in a trial. We all knew immediately that she did not have the placebo. She improved dramatically immediately. And now she is regressing. And there is a lifeline out there that she is not allowed to grab. It breaks my heart. In fact, tears are streaming down my face as I write this. What is the downside to not lowering the age?
Opa

I have the JOY of being Amelia Hamilton's Oma. When Amelia was very young, she kept falling down. It was not long before her family realized that something was wrong. After 3 years of countless hospital and doctor visits and too many tests to count, Amelia was diagnosed with Friedreich's ataxia. Not only does Amelia live with this rare disease, but she also has an aggressive form of FA. I have been a long-time fundraiser for the Alzheimer's Association. When Amelia was diagnosed with FA, I added Friedreich's ataxia to my fundraising as pediatric research is critical. I implore you to allow Amelia and others diagnosed with FA and under the age of 16 to take the FDA drug, SKYCLARYS, approved in February 2023. Time is not on their side.

Elizabeth, as the mother of a child who died from a disease that had no cure, I feel every word expressed in your column. Amelia is truly blessed to have a mother advocating on her behalf and the wellbeing of other children impacted by FA. It is my prayer and hope that pediatric drugs trials will be expedited and open for children like Amelia. I am convinced that there is enough brilliance and knowledge to make this happen. FA children should not have to lose one more thing.

A good treatment today beats a perfect treatment years from now. Let's get this treatment approved for the little one ASAP.

I cannot imagine how difficult it must be to parent and advocate for a child with FA. I wish we could all wave a magic wand, and the medication could be given to all of the patients. The patients need to dance their own dance, and to have some hope and joy.

I’m hoping that approval for under-16-year-olds will happen ASAP!

It's amazing that a treatment has been approved. I am hoping beyond hope that your daughter and other kids like her who are younger than 16 can get started on this lifesaving treatment as soon as possible.

The children are our future! It's critical that we adults help them in order for them to live their best lives every day. The FA treatment should be available to ALL children, not limited by age. During this holiday season, make room in your hearts for them.

Sending big prayers this will be approved sooner rather than later for the pediatric patients. Let’s give these littles the same opportunity.

A mother's love so beautifully captured. These stories are so important. Thank you for sharing!

Elizabeth, thank you for sharing Amelia's journey. Lifting prayers that a cure can be made available for Amelia and others like her very, very soon!

Help must be available for all with FA, regardless of their age. Please listen to the children who have dreams; to the parents who want hope for their children; listen to common sense which dictates that medication which is available, be available for all.

Your strength is inspiring. Praying for Amelia and your family and that pediatric patients can soon receive access to the magic blue pill too. All children should have the same chance - time is of the essence for these little angels!

If this drug developed by the Biogen Corporation is safe and effective, then Biogen need to develop a dosage for children under 16. There is a great need in younger children for this drug.

Thank you for sharing these powerful and heart-wrenching messages about Amelia. Your advocacy for her and all those with Friedreich's Ataxia is inspirational. I hope that a way forward for the necessary testing to get this new treatment to children under 16 can be found and can be expedited as much as possible. Time is of the essence for these precious vulnerable children. Lifting prayers for strength, courage and comfort for all of you.

Elizabeth,

Thank you so much for writing this column. My husband and I were just watching some videos of our daughter Arlee when she was 2 and we were out getting a Christmas tree. We both commented about how stable she is, how she can squat down and stand back up without falling, and how she her walking at age 2 was more composed than at age 7. There are VERY few people who feel that kind of pain and I hate that you have to go through it too. Thank you so much for advocating for our young kids. We so need to get them a treatment as quickly as possible. We hope and pray something is in the works before Arlee and Amelia loose even more than they already have.
Prayers for you family!
God bless

I've had the pleasure of Amelia's company with her beautiful, bright, loving smile and quick wit at family gatherings. It makes the urgency of immediate drug trials on younger children so important that she might live a year or two longer and grace our lives with hers.

Thank you for the informative article regarding this disease and your family. I was left wondering , since 16 year olds can take this medication, why would a 12 year old not qualify. Puberty, not tested on 12 year olds? Is there testing trials on the younger age group?
It’s encouraging to hear of the progress the drug company has made. What are the benefits of a 12 year old taking this approved medication? What are the negatives? Are the benefits for pediatric patients to take this medication greater than any side effects of receiving it? Hope in the future.

This situation is just heartbreaking. It is impossible to understand why a life saving drug is unapproved and unavailable for children! There is no alternative for these little ones, so why?!

Progress for the pediatric patients can’t happen soon enough!

Gut-wrenching read. It is incredible that treatment for adults was approved, but it is devastating that there isn't a 'magic blue pill' available for children yet. This needs to be a priority. I can't imagine a future where we are not able to protect our youngest minds and bodies as they ARE our future. I am hopeful that we can see movement towards this goal.

Thank you for sharing your story. I hope Amelia can gain get the best treatment available.

PLEASE move forward on pediatric trials to hopefully save some of the children! Thank you for your work on this devastating disease!

I'm hopeful biogen will be available for youngsters soon

I spent the first eight years of my career working as an R&D chemist for a pharmaceutical company. As a scientist, I fully respect pharmaceutical companies who are careful and methodical during development for the sake of patient safety. However, when I read stories like this one from Elizabeth Hamilton from people who are experiencing tremendous emotional pain while watching a loved one deteriorate,.it can be challenging to justify a slower, more cautious approach to drug development. My hope is that Biogen may soon be able to move forward with the pediatric studies so that precious children like Amelia may have hope for a future.

My prayers for your family and Amelia. I hope in the near future that Amelia and other like her, will be able to gain access to this hopeful medication.

Powerful. I wish you the best. Thank you for sharing.

I pray that the medication becomes available sooner for pediatrics so treatment can begin to help Amelia with her diagnosis. May God be with you on your journey.

If there is a treatment available it should be used so these children can have hope. The parents and families need to know that research is out there looking for ways to improve those with this disease. Clinical trials should be offered to the children without limits on age. Most of them only have a short lifespan without a treatment to extend their lives.

In this season of hope, let us hope that those in charge of trials will change the minimum age requirements for those children who do not have the luxury of time. Know that others are lifting your daughter, you and all of your family in prayers.

I can't imagine the terror of adults who face terminal illness and know their time is short, let alone children and adolescents who are at the dawn of realizing their circumstances. I would hope that the medical community could "recalibrate" it's stance on trial treatments for those who obviously have no other options, and even less time.
Prayers for parents and families who have rearranged their lives to work tirelessly to advance these treatments.
Prayers for researchers who develop miracle medicines. May they be released to the kids who are running out of time.

Life is full of many mysteries and watching someone you love lose more and more body functionality when a medication is available is a major one. Our girl Amelia is a fighter and so are her parents and sister. Please join with us in hoping medication is soon made available for children with FA. I’m one of the many who love her and lucky me her mother’s mother. Thank you everyone who supports this family.

I don't know Amelia but I know her grandparents and how much they are hoping to share lots of tomorrow's with her. If we could get medication approved for young people like Amelia, we could change so many lives!

Let's pray and push for the approval of this treatment for under-16-year-olds and pray it will happen ASAP!

Please have mercy and give younger children a chance to survive with the new medications available only to those 16 and older!
Dive younger children a CHANCE AT surviving WITH THIS NEW MEDICATION!!!

Thank you so much for sharing your story. First of all, sending all good wishes to you all for a full healing and the hoped for outcome. Secondly, I do understand the need for care and caution in approving new drugs, but I am hoping Biogen will move quickly to get this into trials for pediatric patients like Amelia, and that the FDA sees this and moves expeditiously as well.

As a daughter who lost her mother to a neurologic disease, I can't imagine how much more difficult it would be to watch your child suffer. I implore the drug company to lower the age for treatment. Hope and small forward steps are powerful and impactful.

"One measure of the heart and soul of any society is how it treats its children." (David McCullough, American Historian)

Amelia's struggles, as so sensitively shared by Elizabeth, deserve the same treatment chances as others. And those critical 'heart and soul' actions need to come quickly. She is most worthy. We lift up Amelia and pray for her protection now and for her future.

Time to move this pediatric trial forward. Too many beautiful children need this.

After reading the article and the comments, I agree: A good treatment today beats a perfect treatment years from now, especially when the families are in so much pain. I say try to help whomever you can even if the kids are younger than the ideal age for treatment.

Imploring Biogen to move forward very quickly with treatment that could benefit younger people with FA. Prayers for Amelia and others with FA.

As a mom and physician it is unthinkable to have red tape blocking access to treatment when the clock is ticking. Isn’t it considered emergency use (EUA)? What is the downside? I hope Biogen and the regulators read this article and think of Amelia as their own.

Please consider making this available to younger pediatric patients. Don't really see the downside.

How heartbreaking to have a child too young for the only known treatment! My hope is that in the new year a new decision will be made, and this medication will be available to Amelia and others who are now too young to take it. How wonderful it would be if she could fulfill her dream of jumping off the bottom step but this time to jump into a renewed life.

Beautifully written Elizabeth. As a parent with an 11 year old daughter recently diagnosed with FA, we are also hoping and praying for access to that little blue pill you speak of. We are honestly desperate for it as we watch our sweet girl start to struggle with basic daily tasks like opening her toothpaste. She loves to dance and we are also watching the activity she loves so much become harder and harder for her. While we wait, watching the progression is devastating. We want to be on that lifeboat with you!

You are in our prayers and I’m hoping that approval for under-16-year-olds will happen ASAP!

Elizabeth, thank you for sharing. We very much hope the approval of the medication happens very soon for Amelia and all the children living with FA.

Please approve this medication for children younger than 16, and let's move this forward for all the children suffering from this disease.

It's heartbreaking that relief is so close and yet so far. I hope the pharma companies do the right thing and push for approval for pediatric use!!

Thank you for drawing attention to this! Biogen, please take the necessary steps to get this treatment approved for pediatric use!

Thank you for sharing that beautifully written perspective. As a Dad to two young boys with FA, I feel your pain, struggles, and heartache seeing the physical struggles getting progressively more difficult. The new treatment provides hope, but it's heartbreaking knowing our under-16s kids can't access it ... every month that goes by for them is so important! Let's hope there are spaces for our kids on the lifeboat soon.

Every child deserves a chance for the only known treatment! I hope this medication will be available to Amelia and others who are now too young to take it very soon!

Elizabeth, Thank you for your heart felt family story. My heart is deeply touched. Hopefully your daughter and so many children who are waiting for life saving and life improving medication will have access to it, very soon. Prayers for the children, families, doctors, scientists, and regulators to make this happen.

I am writing in support that this creative, sweet girl get the opportunity to receive a new treatment. As a clinical counselor and previously as an early intervention specialist (as well as the sister and aunt of family with special needs), I know how important it is to have viable resources and HOPE!

I am writing in support that this creative, sweet girl and other young children with FA get the opportunity to try this treatment. As a clinical counselor and previous early intervention specialist (as well as the sister and aunt of family with special needs) I know how important it is to have viable resources and HOPE!

Please give pediatric children the opportunity to benefit from this medication by expediting the trials for younger patients. Time is of the essence for these children so please make this a priority to give them and their loved ones HOPE.

Get this treatment approved for younger children!

Please, please, PLEASE approve this drug. If not now, WHEN?

Biogen, please move forward quickly so that treatment can benefit younger people with FA. All children should have access to the care that is needed!

I had never heard of this disease until now. Having had 1 child with chronic kidney disease ( my husband donated his kidney to him when he was 21) and losing my second son at birth to a similar kidney anomaly, I know how terrifying and difficult having an ill child can be. I will hold you and your family close in my heart and hope that the medication that is so urgently needed will become available immediately. Much love to you.

Hope this gets approved for these beautiful children.

Please know your story is heard and there are legions behind you in this struggle. Sending support for a fast track approval of this drug.

Thank you for sharing your family's story with us. Your daughter is so lucky to have such love and support, but it is so difficult to watch your child suffer when a new treatment might give the gift of time and hope. Surely the approval process to allow younger children to participate in trials for this medication could be expedited, especially when time is of the essence!

Please value all children and initiate a simple dosing trial for FA children under the age of 16. You can increase the lives you are saving and begin answering important questions!!

Early Intervention is a powerful tool to assist childrens' development and this 'little blue pill' appears to be an obvious and necessary tool for young children struggling with FA. Having worked in the field of Early Intervention for almost forty years with very young children, with varying levels of developmental disabilities, I can categorically state that Early Intervention works. The earlier the better. To withhold the only known, proven treatment, waiting until a child is sixteen, is counterintuitive and detrimental. As Emily said, "...Time to move this pediatric trial forward. Too many beautiful children need this."

Thank you for sharing Amelia's story, I hope that treatment options become available very soon for your sweet daughter.

I cannot imagine the frustration of having a child too young for the only known treatment of this devastating disease! I hope and pray the upcoming year will bring change and healing for your beautiful family and that Amelia and others like her will have the chance to dance, play, jump and run again as the carefree children they should be! Please allow this medication to be shared with the younger children.

Good people of Biogen - that's what you are, not a faceless corporation, but a community of folks whose work is invaluable to the everyday lives and literal survival of so many. Please, please, please do whatever it takes to share your healing medicine with those children who are right now, in this moment, suffering. And with all those who will heartbreakingly suffer in the future.

I have full faith that you will indeed because I bet all of you have young people in your lives that you absolutely adore and cherish too!

Thank you, Beth, for enlightening those of us in the ataxia community that Skyclaris is only approved for patients age 16 and older. We need to continue to fight for patients and their families to be empowered to choose to take new medications, understanding the risks but knowing time is critical in stopping this debilitating disease in its track. LIttle Amelia is a fighter, and it's our responsibility as a community to make our voices heard. I will be reaching out to Biogen on behalf of our ataxia support group.

Besides Amelia, I know of another young girl that had this disease. She in fact has died. Please continue your research so that the age group can be lowered to include any child suffering before it is too late for them.

Hoping the drug can be tested and approved for younger children very soon.

Friedreich's ataxia seems one of the most dreadful conditions a child could experience. If there is a way to help the afflicted child, it should be made available as soon as humanly possible. According to Amelia Hamilton's family, Biogen has developed a drug that would literally save Amelia's life--if it can be made available to her soon. Who would not urge them to do so!

I am writing as a friend of the Hamilton family and as a former employee of Biogen. I spent 6 happy years in their Legal Department in the late 80's. I know the company to be innovative, caring, compassionate and on the cutting edge of technology. Please take whatever steps necessary to begin clinical trials for pediatric cases of FA so the families of these children have hope for the future. Please be a leader in the fight against FA for its youngest victims.