14-month old is suffering from rare genetic disease and needs a $2.1 million drug to save her life

A 14-month old baby girl has been diagnosed with a rare genetic disease that can only be treated with a drug that costs more than two million dollars. Ayah Lundt needs this potentially life-saving drug before she turns two. She turned a year old this January and her parents are doing everything they can to get Ayah the treatment she needs. Unfortunately, Zolgensma, the $2.1 million dollar drug has not been approved in Denmark, where the family currently resides. They are raising funds for her treatment in the United States, where Zolgensma was approved in 2019 for use on children under age 2, reported CNN.

Ayah was a healthy baby who grew up meeting all the milestones as expected. She could crawl and even started eating new types of foods. From the picture of perfect health and growth, she started showing worrisome signs of some sort of sickness when Ayah could not even lift her head up while lying down. She was diagnosed with spinal muscular atrophy (SMA.) According to Kids Health, it "is a genetic condition that causes muscle weakness and atrophy (when muscles get smaller.)" It affects a child's ability to crawl, walk, sit up, and control head movements. In severe cases, it can lead to difficulty in breathing and swallowing as well. 

"I think about the cost every day, and it weighs me down," Mary Mithika, Ayah's mother said through tears about Zolgensma which is the world's costliest drug. "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?" The drug has proven to be effective and can save lives with just one dose.  David Lennon, president of AveXis, Inc., of Bannockburn, Ill., the company that developed Zolgensma said, "We saw just remarkable results for these kids," about the many other babies who have received the treatment. He even believes that the medicine is "highly cost-effective" and a "fair and reasonable price" since it's a one-time dose.

The Lundt family has reached out to people through GoFundMe as well as through social media campaigns to raise money for Ayah. The description on the fundraiser stated, "By giving Ayah this treatment, we hope to give her an almost normal childhood where she will be able to play with her big sister without any major obstacle. We are in a race against time to achieve our goal since this treatment is currently only approved for children under 2-years-old." The family has started talks with Boston Children's Hospital for Ayah's treatment once they manage to raise enough money for the drug as well as the additional costs of tests and doctor visits in the US.

"We just want what every parent wants -- the best for their child," Frank Lundt, Ayah's father said. "We were so excited about having another princess. She is perfect," he added. "She has always been such a happy child, even now with all this going on." Ayah has a two-year-old sister, Amira. "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," Mithika said. "Every patient is different. But in just the last few weeks, she's deteriorated so much." Her condition is only getting worse since the diagnosis. She is no longer able to bear the weight of her own body and is supported by a wheelchair.

Ayah is currently being treated in Denmark but is showing no signs of improvement. She gets breathing treatments twice a day, through continuous positive airway pressure, or a  CPAP machine. She is also getting alternative medicine via a lumbar puncture every few months to help build her muscular strength. "She's like a four-month-old trapped in a 14-month-old's body," Mithika said. You can donate for Ayah's treatment here.