Lucas’s story, by his mother
“Because of GM1, Lucas has not been able to grow up with two of his siblings”
How has GM1 gangliosidosis affected your child?
Because of GM1, Lucas has spent over 80 hours flying in an airplane to participate in the gene therapy clinical trial he is enrolled in. Airplane rides have typically not been a pleasant experience for him or those sitting around him! Haha!
During these visits, he has submitted to repeated tests including MRIs, lumbar punctures, audiology exams, X-rays, EEGs, nerve conduction tests, developmental assessments, eye exams, stomach ultrasounds, and heart tests. Lucas has not been able to understand why he was asked to participate in these tests, and cooperation has been difficult for him. He has undergone all of this to assess the safety of the gene therapy drug, for the chance of an improved quality of life for himself, and to help make possible a future treatment, if not cure, for all GM1 children.
How does GM1 impact your family?
Because of GM1, Lucas has not been able to grow up with two of his siblings. His older brothers, Eli and Evan, passed away at the ages of 11 and 10 (in 2020 and 2022) when he was just a few months old and two years old. Because of GM1, Lucas’s oldest sister, who is now 17, has grown up with a lot of stress in the home with her younger brothers needing constant care and frequent medical attention.
GM1 has not been all bad. Despite the difficulty and strain it has put on our family, it has been an eye-opening experience that has taught us compassion for the many challenges and suffering people around the world may experience. It has also given us opportunities to connect with so many in our communities. These people have lifted us and impacted our lives for good.
What do you wish people understood more about rare diseases?
I wish people understood that it’s okay to reach out and get to know individuals affected by rare diseases, just as they would get to know anyone else. It’s okay to ask questions to understand what their individual needs and struggles are.
Reaching out helps the community understand the situation better and then hopefully inspires them to rally around the family with support. In my experience, families are willing to share their experiences and accept help from people who genuinely care.
Why should people support the Cure GM1 Foundation and rare diseases?
The Cure GM1 Foundation works hard to bring the GM1 community together; inspires doctors, specialists, and entrepreneurs to work toward a cure; and helps provide hope and purpose for families living with GM1 gangliosidosis.
We are grateful for the positive impact the Cure GM1 Foundation has had in our lives!