A 14-month-old who was diagnosed with a degenerative disease desperately needs a drug that could potentially save her life. There is only one caveat— the treatment costs $2.1 million per dose.
Denmark toddler Ayah Lundt was diagnosed with spinal muscular atrophy type 2 (SMA) in November, a rare genetic disease found in 1 in 10,000 children across the globe. The disease kills nerve cells and causes infants' muscles to deteriorate, which can lead to difficulty swallowing or breathing. Typically, children suffering from SMA do not survive past early childhood due to respiratory failure.
Now, her parents are racing to secure enough money to provide Ayah with the one-time treatment drug known as Zolgensma, all before her second birthday.
Ayah's diagnosis came after a seemingly healthy baby began to exhibit alarming symptoms.
After her January 2020 arrival, Ayah was in perfect health in the early stages of her life. Within the first eight months, Ayah was eating, walking and hitting all of her usual developmental milestones.
It wasn’t until she was around nine months old, that her parents noticed their daughter was having trouble doing the things she had previously been capable of.
Ayah’s legs caved when she tried to crawl or stand and she could no longer lift her head while lying down, sit on her own or clap. Her Kenyan mother, Mary Mithika, grew increasingly worried when she noticed the 14-month-old was unable to eat oatmeal with a spoon, an act that she had enthusiastically done before.
Due to issues with Zolgensma, a gene therapy treatment, in Denmark where the family currently resides, Mithika and her husband, Frank Lundt, have pivoted their efforts to the United States, where the drug was approved by the FDA in 2019 to be used on children under the age of 2.
"I think about the cost every day, and it weighs me down," Mithika said through tears, CNN reported. "And then I look at Ayah and see her getting worse. As a parent, what would you do if you knew there's something out there that can save your child?"
Mithika said that Ayah will never walk again, but that doesn't mean there isn't still hope.
Treating and caring for a patient with SMA could “cost up to $6 million in the first 10 years of a patient’s life,” Novartis Gene Therapies president Dave Lennon told CNN.
The drug is also not a guaranteed cure, but the powerful intravenous dose could keep Ayah breathing, altering her genetic expression and improving muscle function.
Due to economic and financial setbacks exacerbated by the pandemic, the family has only been able to raise around $60,000 USD as of late March via GoFundMe.
"We just want what every parent wants — the best for their child," the toddler’s Danish father said. "We were so excited about having another princess. She is perfect."
"She has always been such a happy child, even now with all this going on," he added.
Ayah also has a 2-year-old sister named Amira.
Over the last few months, Ayah’s condition has worsened, forcing her to become wheelchair-bound.
"She's like a four-month-old trapped in a 14-month-old's body," Mithika said. "She gets frustrated she cannot move. When her older sister dances around her, she tries to join her from the wheelchair but she can't. Every patient is different. But in just the last few weeks, she's deteriorated so much."
In addition to the GoFundMe, Ayah’s parents have also launched a social media campaign titled “Friends of Little Ayah,” in an effort to share her progress with the world and encourage people to donate in the fight to save her life.
