July 9, 2021 -- Athletes, influencers, musicians, and people around the globe are teaming up in a final push to raise money that could potentially save the life of a 17-month-old girl in Denmark.

At 10 months old, Ayah Lundt was diagnosed with type 2 spinal muscular atrophy (SMA), a rare genetic disease that affects about 1 in 10,000 children. Children and babies with SMA often have trouble breathing, swallowing, controlling their head movements, and sitting up without assistance due to the weakening and shrinking of their muscles due to a defective or missing gene.

SMA is the No. 1 genetic cause of death in infants. The good news is there is a treatment. The bad news is the medication costs a whopping $2.1 million.

The FDA approved the drug Zolgensma in 2019 to treat SMA in children under the age of 2.

More than 1,200 patients have been treated with Zolgensma globally, a spokesperson from Novartis Gene Therapies, the company that produces the drug, says in a statement. In the United States, there are a number of insurers that cover Zolgensma, sometimes with certain limitations.

In Denmark, Zolgensma is only approved for children with SMA under 6 months old, even if a patient over 6 months old can afford the drug.

Because Ayah is ineligible, her parents, Frank Lundt, who is Danish, and Mary Mithika, who is from Kenya, say they are in contact with Boston Children’s Hospital about treating Ayah if they raise enough funds to cover Zolgensma and travel arrangements.

After her diagnoses, Ayah’s family began raising money to afford the medication before Ayah turns 2. Mithika says they raised around $60,000 in 4 months, which only scratched the surface of the money they need.

When CNN published an article on Ayah’s story in late March, the family raised $1 million dollars in 24 hours, according to Mithika.

“We did not sleep,” Mithika says. “We were literally scrolling on GoFundMe, refreshing every minute up until the morning. We were like, ‘Oh my God, pack your bags, we're going to the hospital next week!’”

However, donations dwindled after a couple of days.

Support Across the Globe

Mithika created an Instagram account to both document Ayah’s journey and drive donations. Some public figures have taken notice. Kenyan musician King Kaka recently went on Instagram Live with Mithika to share Ayah’s story with his more than 1.5 million followers.

Actor and author Teresa Palmer also joined Mithika for an emotional Instagram Live session. Palmer recounted the first time she saw Ayah’s picture, before both Palmer and Mithika broke down into tears.

Liverpool soccer player Divock Origi is just one athlete who has auctioned signed jerseys to donate to Ayah’s cause.

On June 26, Ayah’s family kicked off “Run for Ayah,” a virtual race through JustMove. Participants can run, walk, or bike a distance of their choosing, with fees going toward Ayah’s treatment. The race ends July 31. There are participants registered around the world, including the U.S., Qatar, Sweden, the Netherlands, and Canada.

A Revolutionary yet Expensive Drug

Zolgensma is the first gene therapy approved to treat children younger than 2 with spinal muscular atrophy.

The price of a drug, however, is not exclusively tied to its benefits or capabilities, according to Erin R. Fox, PharmD, senior pharmacy director at the University of Utah Health. Research and development of a new drug can cost billions of dollars.

“There are no requirements for a product to be priced according to therapeutic benefit,” Fox says in a statement. “Basically, new products are priced at whatever the market will bear.”

In just one dose, Zolgensma replaces the missing or defective gene that causes SMA with a working copy, allowing some patients to build up enough muscle strength to lead otherwise normal lives.

The cost of the drug, however, still makes it out of reach for many patients.

Hope for the Future

Ayah is unable to crawl, roll over, sit-up straight, or control her head movements, all things she had been able to do before her muscles started weakening at 9 months old, according to Mithika.

Mithika said she keeps her spirits high with the help of “Friends of Little Ayah,” an endearing term for organizers around the globe passing out flyers, online fundraising, and donating their unique talents in efforts to raise awareness.

“There are people who literally don’t sleep at night fighting for Ayah,” Mithika said during one Instagram Live session. “How could I ever give up?”

WebMD Health News

Sources

Forbes: “At Over $2 Million Zolgensma Is The World's Most Expensive Therapy, Yet Relatively Cost-Effective.”

Medicinraadet: “The Danish Medicines Agency recommends Zolgensma for infants with spinal muscular atrophy.”

Zolgensma.

Novartis: “Novartis Financial Results – Q1 2021.”

Business Insider: “Aetna and Anthem will now pay for more kids with a devastating rare disease to get a $2.1 million drug, reversing earlier denials.”

BioPharma Dive: “Zolgensma set a new drug pricing bar. Insurers show some signs of pushback.”

Journal of the American Medical Association: “Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018.”

News release, FDA.

CNN: “She's 14 months old and needs a drug that costs $2.1 million to save her life.”

Erin R. Fox, PharmD, senior pharmacy director at the University of Utah Health and board-certified pharmacotherapy specialist. 

JustMove: “Run for Ayah.”

FDA: "FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality."

GoFundMe: " Together for Little Ayah."

JustMove App: "Run for Ayah."

 

© 2021 WebMD, LLC. All rights reserved.
Click to view privacy policy and trust info