The moment we received the news, my five-year-old daughter, Lucy, looked at me with concern through her bright pink glasses. “Why are you crying, Mommy?” she asked.
“I’m worried about your medicine,” I explained. “We don’t know how we will get it soon, and if you can’t get your medicine, you will get sick.” She paused, and with a look of deep trust, she replied, “I’m not sick, Mommy. I won’t be sick.”
I wish more than anything that could be true.
When I say “get sick,” I’m using a euphemism for a harsh reality: without her medication, she could die. How do you explain to a five-year-old that they are slowly fading away? That they suffer from a relentless, fatal disease, and the only treatment sustaining her life will no longer be available come October? This effective medicine exists; it just won’t be given to Lucy and many other children like her. A decision made somewhere has dire consequences, and my daughter will pay with her life.
Lucy is a vibrant five-year-old with an unyielding love for life. She adores wearing her pink heart sunglasses over her regular glasses, twirling in her tutu, and belting out “Let It Go.” Her favorite show is Pioneer Woman, and she often wonders aloud, “I wonder where Ladd is on the ranch today.” She dreams of a future filled with possibilities: getting married, becoming a doctor, and following her big sister everywhere.
However, Lucy also battles a rapidly progressive and terminal illness known as Niemann-Pick Type C (NPC), sometimes referred to as Childhood Alzheimer’s because it can lead to dementia in young children. It causes seizures, tremors, and choking. But the most heart-wrenching aspect is not just what this disease inflicts, but what it steals away: her laughter, her memories, her songs, her very life. Instead of birthday parties and dance recitals, we face suction machines, feeding tubes, and a mini hospital setup in our living room. Ultimately, it will take the most precious thing of all: her life.
This is what her medication, VTS-270/Adrabetadex, is fighting against. Thanks to this treatment, Lucy is currently stronger than the NPC, but that balance is precarious. In January 2021, the pharmaceutical company, Mallinckrodt Pharmaceuticals, announced the discontinuation of all access programs and distribution of the drug by October 2021. Time is running out; we have only six months left.
Lucy receives this medication every other week through a lumbar puncture, a procedure she bravely endures while fully awake at the remarkable Children’s Hospital Los Angeles. She is cheered on by some of the most compassionate doctors and nurses, who make every visit magical with their matching pink glasses and unicorn pins. They recognize the importance of this drug and advocate for its continued access.
Niemann-Pick Type C patients have safely used VTS-270/Adrabetadex since 2010. Lucy began her treatment at 19 months old in 2017 through a compassionate use program. Before starting the treatment, she struggled to walk, was very weak, and choked on soft foods. We were uncertain if she would see her second birthday due to the aggressive nature of her condition.
Then, something miraculous happened. After just five doses of VTS-270, she began to walk and thrive. Today, she’s in pre-K, rides a tricycle, and climbs into her sister’s top bunk bed. This medicine has given her a chance at life, and without it, she will lose everything.
The reasons why this medication hasn’t achieved full approval are complex and convoluted, making it one of the more challenging stories in drug development. But the reality is clear: without this medicine, Lucy will die. And so will countless other children like Emma in Minnesota, Jeg in Illinois, and Abby in Texas.
As a mother, I don’t seek to assign blame; my focus is on urgent action. This medicine works, and her doctors can confirm its effectiveness. I am fighting for my daughter’s life and the lives of the many others who depend on this treatment for survival. We need to preserve a medicine that allows Lucy to experience childhood, forge connections, and find joy in life.
This is a solvable issue. We can ignite the necessary changes to save this medicine. But our children need your support. We call on all mothers, an army of advocates, to stand for our children and help us secure their future. Together, let’s ensure they have a chance to grow up, trust the world around them, and live fulfilling lives.
For more insights on this vital mission, connect with us on Facebook and Instagram. To help fund the expert assistance we need to navigate these complex issues, please check out our campaign “Save Our Medicine”.
If you’re interested in additional information on home insemination, check this blog post, or visit Make a Mom for expert resources. You can also find valuable insights about fertility here.
Search Queries:
- home insemination kit
- self insemination tips
- home insemination methods
- artificial insemination at home
- best home insemination kit
Summary:
A mother’s heart-wrenching struggle reveals that her daughter Lucy’s life depends on a medication that is set to be discontinued in six months. Lucy, who suffers from a fatal disease called Niemann-Pick Type C, has thrived thanks to this drug, but the pharmaceutical company has announced its withdrawal. Desperate for action, the mother calls upon others to help save Lucy and other children who rely on this treatment for survival.
Leave a Reply