For the first few months of her life, Ayah Lundt was the picture of health.
Born in January last year at a whopping 8.3 pounds, the bubbly baby girl with dark curls and bright brown eyes hit all her developmental milestones.
By six months, she was crawling and trying new foods. Bananas and mushy broccoli were her favorites. Avocados, not so much.
Then at nine months, her progress suddenly reversed. She could not lift her head while lying down, sit on her own or clap — all things she’d excitedly done before.
Her legs caved when she tried to crawl or stand. The key red flag came when she was unable to eat oatmeal with a spoon — something she’d done numerous times.
For a baby who loves her food, that was especially worrisome, her mother, Mary Mithika, told CNN by phone from their home in Bornholm, Denmark.
Ayah was diagnosed with spinal muscular atrophy, a genetic disease found in one in 10,000 children born worldwide.
She’s now 14 months old and her parents are in a race against time to raise $2 million for a one-time, potentially life-saving treatment before she turns 2.
The treatment is called Zolgensma. At $2.1 million (Ksh.230million) a dose, it’s referred to as the world’s most expensive drug.
“I think about the cost every day, and it weighs me down,” Mithika said through tears before taking a break to compose herself. “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?”
Spinal muscular atrophy (SMA) is a progressive disease caused by a defective gene. The disease kills nerve cells and causes infants’ muscles to waste away, which can lead to difficulty swallowing or breathing.
As their muscles deteriorate, infants suffering from SMA develop finger tremors and a curved spine. They cannot stand or walk unaided, and most don’t survive past early childhood due to respiratory failure.
Zolgensma, the gene therapy treatment, is not a guaranteed cure. Ayah’s mother says she will never walk again.
But a one-time intravenous dose delivers a fully functional copy of the gene into the target nerve cells, altering the child’s biology and improving muscle movement and function.
The drug is not approved under universal health care in Denmark, the small Scandinavian nation of about 6 million people.
So Ayah’s parents are raising money to get her treated in the United States, where Zolgensma was approved in 2019 for use on children under age 2.