The moment we learned the news, my five-year-old daughter, Lily, looked up at me with concern in her bright pink glasses. “Why are you crying, Mommy?” she asked.
“I’m worried about your medicine,” I replied. “We don’t know how we will get it soon, and if you can’t get your medicine, you will get sick.” She gazed at me for a moment, and with an unwavering sense of trust, she assured me, “I’m not sick, Mommy. I won’t be sick.”
I wish that could be true.
The phrase “get sick” is a euphemism for a harsh reality: she could die. How do you explain to a five-year-old that she is slowly deteriorating? That she has a relentless, life-threatening condition and that the only treatment keeping it at bay will no longer be available after October? This medication is effective and safe, yet it will no longer be administered to her or dozens of other children like her. Someone made the decision to discontinue it, and now she may pay for that choice with her life.
Lily is a spirited five-year-old full of dreams and laughter. She adores wearing her pink heart sunglasses over her regular glasses, dancing around in a tutu, and belting out “Let It Go.” Her favorite show is “Pioneer Woman,” and she often wonders, “I wonder where Ladd is on the ranch today.” She dreams of getting married, becoming a doctor, and following her older sister around for the rest of her life.
However, she also battles a rapidly progressing neurodegenerative disease known as Niemann-Pick Type C (NPC), often compared to Childhood Alzheimer’s due to its potential to cause dementia in young children. It leads to seizures, tremors, and choking. Yet, what’s most cruel is what this disease robs her of: her laughter, her memories, her ability to dance, and ultimately, her life.
This is what her medication, VTS-270/Adrabetadex, is fighting against. Thanks to this treatment, Lily is currently stronger than the NPC, but time is running out. In January 2021, Mallinckrodt Pharmaceuticals announced the termination of all access programs and distribution for the drug in October 2021. We have just six months left.
Lily receives this medication biweekly through a lumbar puncture, which she bravely endures at the renowned Children’s Hospital Los Angeles. Each visit, she is surrounded by exceptional doctors and nurses who cheer her on and bring magic into her life. They recognize the profound impact this drug has on her well-being and advocate fiercely for continued access.
Since 2010, Niemann-Pick Type C patients have safely used VTS-270/Adrabetadex. Lily started her treatment at 19 months old, through a compassionate use program. Before starting, she struggled to walk, was frail, and had difficulty swallowing. We feared she wouldn’t make it to her second birthday due to the aggressive form of NPC she has.
Then, a miracle happened. After just five doses of VTS-270, she began walking and flourishing. Today, she is in pre-K, rides a tricycle, and even climbs into her sister’s top bunk. This medication has given her life, and without it, her future is uncertain.
The complexities surrounding the approval of this drug have been described as one of the most intricate stories in drug development. While the reasons are convoluted, the reality is stark: without this medication, Lily will not survive. That fate also looms over other children, like Mia, a two-year-old in Minnesota, and Sam, a seven-year-old in Illinois.
As a mother, I’m not focused on assigning blame; I just want a solution. This medicine is effective, and her medical team recognizes its importance. My priority is to fight for Lily’s life and for the many others who depend on this drug. It’s about ensuring that children like Lily can experience joy, love, and connection. She deserves to live and thrive, not to lose faith in the world around her.
This is a solvable issue. We can drive change to save this medication, but we need your support. We call on all mothers to unite for our children’s futures. Please help us advocate for access to this vital medicine and give our children the bright futures they deserve. They are counting on us; let’s guide them away from despair and toward hope.
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Summary
My daughter Lily’s life is at risk due to the impending discontinuation of her crucial medication, VTS-270/Adrabetadex. As a five-year-old battling Niemann-Pick Type C, a devastating neurodegenerative disease, she relies on this drug to maintain her strength and quality of life. Despite its effectiveness, the pharmaceutical company plans to halt distribution in six months. As her mother, I urge all to join us in advocating for access to this lifesaving treatment, ensuring that children like Lily can continue to experience joy and connection in their lives.
- What is Niemann-Pick Type C?
- How does VTS-270/Adrabetadex work?
- What are the symptoms of NPC?
- How can I support NPC research?
- What are the treatment options for NPC?
Keyphrase: Niemann-Pick Type C medication discontinuation
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