It was when she was nine months old that little Ayah Lundt's parents began to notice strange behavior.
Despite having been a healthy baby from birth, the baby could not lift her head when lying on her stomach, nor could she sit up with her own efforts or use her hands to clap her hands.
Her mother, Mary Mithika, also noticed that the baby's little legs had no strength when she tried to crawl or stand up.
The baby also stopped eating oatmeal with a spoon, something she had done numerous times.
The family, who resides in Bornholm, Denmark, went to the doctor, where little Ayah was diagnosed with
Spinal Muscular Atrophy
(SMA), a rare genetic disease that affects one in 11,000 children born worldwide.
[When can children get the COVID-19 vaccine? And will it be recommended?]
The terrible news was accompanied by an even more painful one:
the treatment for this disease, called Zolgensma and made in the United States, costs more than two million dollars
and is considered the most expensive in the world.
Ayah has another sister, two years old.
Verner Verner Kjærsgaard
Now the baby is 14 months old and her parents are in a painful race against time to raise the large sum to pay for the treatment that could save her life, before she is two years old.
"We only want what all parents want: the best for their children," Fran Lundt, the baby's father, told CNN television.
Ayah has another sister, Amira, two years old.
"We were so excited to have another princess. She is perfect," said the father.
"She has always been such a happy child, even now with everything that is going on."
Zolgensma, considered the most expensive drug in the world, is a treatment for children suffering from SMA and the most revolutionary for treating the disease in children under two years of age.
But since it was introduced in 2019 in the United States, it has been accompanied by controversy, especially due to its exorbitant price: 2.1 million dollars.
Vas Narasimhan, chief executive of Novartis, the drugmaker that makes Zolgensma, has defended the price of the drug since its launch, targeting other areas of medicine that have high prices.
"Critics are not thinking about how our health systems work," Vas Narasimhan said in 2019. "For transplants, we spend between three and five million dollars [per patient] much less effectively than we see with a drug like East.
We are trying to price it at 50% of what is the current cost of care for ten years for these children. "
Ayah's mother sings songs to her while applying palliative treatments that are uncomfortable for the baby.
Charles Njuguna
According to Narasimhan, the drug has worked for all patients who have received it.
"In our first studies we had 15 children who were followed for about four years and all the children are alive and doing well," he said.
Zoelgma displaced what was until then the most expensive medicine in the world: Luxturna, a drug that sells for $ 850,000 and is used to treat blindness caused by a rare inherited disease.
And there is no hope that their price will drop, as there is no incentive for pharmaceutical companies developing treatments for rare diseases to charge less for these therapies: they often have no competition.
[Cases and severity of childhood syndrome linked to COVID-19 increase. And Latinos are the most affected]
The race to get the funds to save Ayah has spread to several continents and a large group of volunteers is trying to unite efforts, but they are not enough yet, probably due to the large sum and the last months have been marked by the financial difficulties stemming from the COVID-19 pandemic.
As of the end of March, they
had only raised about $ 60,000.
The therapy is approved for use in at least 35 countries, but Denmark is not one of them.
The Nordic country is evaluating whether its cost should be reimbursed under the universal health care system prevailing there.
A nurse manages to bring hospitalized babies closer to their parents through WhatsApp
March 29, 202102: 41
According to the US National Library of Medicine, SMA "is a genetic disease that attacks nerve cells called motor neurons found in the spinal cord."
These neurons communicate with voluntary muscles, that is, those that people can control, such as those in the arms and legs.
As muscles lose neurons weaken.
This can affect the ability to walk, crawl, breathe, swallow, and control the head and neck.
SMA has a family trend, according to doctors.
Parents usually have no symptoms, but they carry the gene.
"She gets frustrated because she can't move
," the mother told CNN. "When her older sister dances around her, she tries to follow her from the wheelchair, but she can't."
Ayah has SMA type II, which develops in babies between the ages of six and 12 months.
Mithika says that, in recent weeks, the baby's health "has deteriorated a lot."
Type I is the most aggressive, since it is very rare that the little ones who suffer from it reach the age of two.
Types II and III also have a reduced life expectancy, because by not being able to move the muscles well or completely, they atrophy.
Type IV is seen in adulthood, after 30 years.
Increase in the number of children with Multisystemic Inflammatory Syndrome, a condition linked to COVID-19
March 7, 202100: 24
In the absence of the treatment she needs, the family is applying palliatives that are painful and uncomfortable for Ayah.
At home, the little girl receives respiratory treatments twice a day.
His parents use continuous positive airway pressure therapy, informally known as the CPAP machine, as a preventative form of therapy on their lungs.
When the baby becomes restless, her mother sings a song to her while placing the respiratory system over her nose and mouth.
Ayah also receives alternative medicine through a lumbar puncture
every several months.
They also try to help her do activities like crawling to strengthen her small muscles.
With information from CNN and NBC News
