More than 20 children live in a race against the clock Spinal muscular atrophy (SMA) in Ecuador. Families suffer knowing that there are drugs to save their children, but they are extremely expensive, exceed $2 million. According to several parents, the procedures established by the state to access the drugs seem “designed to sentence these babies to death.” Cases of O Y Michael are proof of that.
Laura Varela and her husband longed to have a child, but life did not give them the opportunity. The woman suffered two losses until one day she received the long-awaited positive. After an uncomplicated pregnancy, Theo was born on March 6, 2022.
At first everything went well, the baby moved his limbs normally. At almost three months old, his mother noticed something was wrong. When he was placed on his stomach, he did not raise his head. After some time, he took him to a neuropediatrician, who told him that it was necessary to perform a exam that is held only in the United States.
Theo was soon confirmed to have spinal muscular atrophy, a disease that damages and kills motor neurons. But the baby suffers from type 1, the most serious and common, which requires emergency treatment, because average life expectancy is two years. Crossroads: The medicine Theo needs is the most expensive in the world.
“For them, every passing day is uncertain because they are losing the strength of their muscles. It is devastating for us to accept the fear that the decision as parents is not in our hands. They took away our chance to save our children,” said Laura Varela.
The family is from Ottawabut constantly travels to Quito because the child is aimed at Baca Ortiz Hospital. Laura admits she feels affected, she says in order to secure the public health system Zolgensma -medicine of 2.1 million dollars– a long process is required that could cost your child’s life.
After the diagnosis of the disease, the medical board of the hospital must meet to analyze the treatment and medication that the patient needs. Then issue an annex (type of prescription). The Department of Health must then process the request. All processes take months and even years, according to testimony collected for this report.
“It is urgent and they already know why they went through these cases. The government should direct the administrative part of this drug in a shorter way,” said Varela.
He adds that with one dose of Zolgensma the disease can stop. “If Theo gets the drug tomorrow, the life expectancy will be over 50 percent. I would even have the opportunity to walk little by little, but control the condition in time”.
The mother doesn’t want the same thing to happen to Derek and other children who didn’t get the medicine on time. Theo currently remains stable and does not require intubation, but the picture may soon change as these babies who undergo tracheostomy (opening in front of neck to aid breathing) they no longer have access to Zolgensma.
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THEY REFUSED THE MEDICATION
Maikel served a year in bed at Carlos Andrade Marin Hospital. He’s been there for three months after getting a lung infection that led to a tracheostomy. He also has stomach tubes to feed himself.
Paulina Pilko, the baby’s mother, remembers realizing a month after his birth that her son could not move his limbs normally. The doctor was puzzled that the baby’s tongue was trembling. The little boy was referred to a genetic doctor who recommended that he be tested for muscular atrophy in the US. This is another expense that parents have to bear, no government hospital can bear it. Only the exam is worth it 800 dollars.
At six months old, Michael was confirmed to have SMA type 1. “everything came crashing down”says Paulina, remembering the day the results arrived. At this point the viacrucis began to receive treatment. The family travels every month from Puyo to the capital to find out if the process had already started, but there were no answers.
Given the hospital’s inaction, they sent a letter to the president, William Lassoand the then Minister of Health, Ximena Garcón. They didn’t answer them either.
The next step was to file a protective action which won at first instance. At this point Michael was breathing on his own. The judge ruled that the Andrade Marín Hospital must assumes responsibility for all damages to delay treatment, assist with apparatus and even medication excuse me public.
However, when the baby was about to turn nine months old, he was admitted to the Emergency Department Baca Ortiz Hospital because during a bus ride he got a lung infection. He was in intensive care for three weeks.
The trial continued. Andrade Marin appealed the decision and at second instance they were denied treatment. By this time the baby had already had a tracheotomy.
“They said the baby was no longer fit, that the children should not use a respirator”, mentions Paulina Pilko. He adds that despite being denied the world’s most expensive drug, the judge ordered the hospital to supply an electronic ventilator and additional supplies.
The parents will continue to fight to save their son’s life and will appeal the judge’s decision. “We are sure that our baby will be fine because there are cases where when he has a tracheostomy, they are given the medicine and he recovers. We hope that this will happen with our baby as well”, he points out. Fernando PiscoMichael’s father.
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Mikel is now stable, no longer having any infections, but remains in the hospital until he is put on a ventilator. When that happens, I would go back to Puyo. His father and his two brothers, who have returned due to lack of funds, are waiting for him there. Meanwhile, Paulina sleeps in a hospital chair.
“It was a very difficult change. I’m here with my baby, my kids over there. Every doctor who examines him tells me that I better pray to God to take him soon. They better help me with the treatment”, says the mother through tears.
He adds that the baby is nervous, has gained weight and has had no complications. “If they put the medicine on him right now, he will recover, he will start moving his body. They are very unfair, they don’t want to help us“.
Fernando has a job for which he receives the basic salary, but he also sells sheets to get more income and cover the costs of his son’s illness. In addition, the family organizes activities such as raffles or solidarity meals, but nothing is enough.
Theo and Michael’s families have launched social media campaigns to get the two million dollars they need to buy Zolgensma, as well as pay for additional expenses: travel, drugs, therapies, devices, etc.
Those who wish to cooperate with Oyou can contact the mother at the following phone number 0967595863. Or deposit in Banco del Pichincha Savings Account # 2203527162 in the name of Laura Varela. Social security number: 1003549183.
To cooperate with Michael and his family, you can find at no 0979498421. Or deposit in Savings Account No. 5976150200 at Banco de Pichincha in the name of Pilco Popayan Paulina Magali.
WHAT IS SPINAL MUSCULAR ATROPHY?
There are about 7,000 rare diseases in the world. One of them is Spinal muscular atrophy (SMA)a group of genetic diseases that damage and kill the motor neurons that control movements of the arms, legs, face, throat and tongue.
As neurons die, muscles begin to weaken and atrophy. It is a progressive wear and tear that affects speech, walking, swallowing and breathing.as described by the MedlinePlus portal.
It has a prevalence of one to two cases per 100,000 people and an incidence of 1 in 10,000 live births. According to the Ecuadorian Foundation for Spinal Muscular Atrophy (FAME), there are 23 patients suffering from the disease in the country.
There are three FDA-approved treatments Food and Drug Administration (FDA) depending on the patient: Spinraza, Evrysdi and Zolgensma. All exceed hundreds of thousands of dollars, except for the last one, which is considered the most expensive in the world with a value of 2.1 million dollars.
Elizabeth Noguerapresident of the Ecuadorian Foundation for Spinal Muscular Atrophy (FAME), states that although these drugs are not part of the National Drug Table, “It is the duty of the government to provide drugs for this class of diseases”.
He adds that last year they had unsuccessful meetings with the then Minister of Health, Ximena Garcónwho promised to sign agreements to expedite the care of minors, but nothing happened. “These are children dying daily, we already lost five last month”.
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LIFE OR DEATH
Noguera also has a son with SMA grade 2. She describes her struggle as terrible. Initially, there was no life expectancy for her little boy, much less a treatment. Then came the joy that the FDA approved the drug.
Daniel is 12 years old, but you have not used the medicine for more than five years. The request was repeatedly rejected by Baca Ortiz Hospital.
“Without medicine, the disease impairs every function. What the government does is wait for time to pass for the children to degenerate and then say they are no longer viable children“.
He adds: “If the children do not receive the medicine, they are sentenced to a cruel death. I don’t know how the government can sleep easy knowing that its people are dying for lack of medicine. Because it is a rare disease, they shamelessly told us that they would rather save 30 children with cancer than one with spinal muscular atrophy”.
Elizabeth Noguera’s son Daniel has type 2 SMA and has been struggling to get his medication for 5 years.
The Foundation calls the Minister of Health, Joseph Rualesto meet with the families and sign contracts for the purchase of medicines, as soon as possible.
Vistazo requested an interview with the Department of Health within a week, but had not received a response as of press time.
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