A race against the clock lives more than 20 children with Spinal Muscular Atrophy (SMA) in Ecuador. Families suffer knowing that there are medicines to save their children, but they are extremely expensive, exceeding 2 million dollars. According to several parents, the procedures established by the State to access the medicines seem ‘to be made to sentence these babies to death.’ The cases of Theo and Maikel are proof of this.
Laura Varela and her husband longed to have a child, however, life did not give them the opportunity. The woman herself suffered two losses, until one day she received the long-awaited positive. After an uncomplicated pregnancy, on March 6, 2022, Theo was born.
At first everything went well, the baby moved his limbs normally. Nearly three months old, her mother noticed that something was wrong. When she turned him upside down, she wouldn’t lift his head from her. Some time later, she took him to a neuropediatrician, who told him that it was necessary to perform a test that is only done in the United States.RELATED
It was soon confirmed that Theo has 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 urgent treatment, since the average lifespan is two years. The crossroads: The medicine Theo needs is the most expensive in the world.
“For them, each day that passes is uncertain because they are losing the strength of their muscles. It is devastating for us to assume the fear that the solution as parents is not in our hands. They have taken away the possibility of saving our children,” said Laura Varela.
The family is from Otavalo, but constantly travels to Quito because the child was referred to the Baca Ortiz Hospital. Laura confesses to feeling afflicted, she says that for the public health system to provide Zolgensma -medication of 2.1 million dollars- a long process is required that could cost her son’s life.
Once the disease is detected, the hospital’s medical council must meet to analyze the treatment and medication that the patient needs. Then, issue an annex (a kind of prescription). Then, the Ministry of Health should process the request. All processes last months and even years, according to the testimonies collected for this report.
“This is urgent and they already know why they went through these cases. The Government should channel the administrative part of this medicine in a shorter way,” Varela said.
He adds that with a single dose of Zolgensma, the disease could stop. “If Theo receives the drug tomorrow, we would have a life expectancy of more than 50 percent. He would even have the possibility of walking little by little, but controlling the condition in time ”.
The mother does not want the same thing to happen that happened with Derek and other children, who received the medicine out of time. At the moment, Theo remains stable and has not required intubation, but the picture could change soon, as those babies who undergo tracheostomy (opening in front of the neck to help breathing) are no longer allowed access to Zolgensma.
THEY REFUSED THE MEDICATION
Maikel turned one year old in a bed at the Carlos Andrade Marín Hospital. She has been there for three months after acquiring an infection in the lungs, for which they performed a tracheostomy. Also, she has gastric tubes to feed herself.
Paulina Pilco, the baby’s mother, remembers that a month after he was born, she realized that her son could not move his limbs normally. She was puzzled by the doctor that the baby’s tongue trembles. The little boy was directed to a geneticist doctor who recommended that he be tested for muscular atrophy in the United States. This is another expense that parents must bear, no public hospital can cover it. The exam alone costs $800.
Six months later, Maikel was confirmed to have type 1 SMA. “Everything fell apart for us,” says Paulina, remembering the day the results came back. At that moment the viacrucis began to obtain the treatment. The family traveled every month from Puyo to the capital to find out if the process had already started, but there were no answers.
Given the inaction of the hospital, they sent a letter to the president, Guillermo Lasso, and to the then Minister of Health, Ximena Garzón. They didn’t answer them either.
The next step was to file a protection action that they won in the first instance. At that moment, Maikel was breathing on his own. The judge ruled that the Andrade Marín Hospital had to take responsibility for all damages due to the delay in treatment, help with the ventilator and medicines, and even apologize publicly.
However, when the baby was about to turn nine months old, he was admitted to the Emergency Room at the Baca Ortiz Hospital because he acquired a lung infection on a bus trip. He spent three weeks in intensive care.
The legal process continued. Andrade Marín appealed the decision and in the second instance they were denied treatment. By that time, the baby had already had a tracheotomy.
“They said that the baby is no longer fit, that children do not have to use a breathing apparatus,” says Paulina Pilco. She adds that, despite the fact that they were denied the most expensive medicine in the world, the judge ordered the hospital to deliver an electronic ventilator and complementary 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 improve because there are cases in which, being with the tracheostomy, they give them the medicine and he is recovering. We hope that the same will happen with our baby”, says Fernando Pisco, Maikel’s father.
Currently, Maikel is stable, he no longer has any infections, but he remains in the hospital until he is given the ventilator. When that happens, he would return to Puyo. His father and his two brothers, who returned due to lack of resources, are waiting for him there. Meanwhile, Paulina sleeps in a hospital chair.
“It has been a very hard change. I’m here with my baby, my children over there. Every doctor who reviews him tells me that I better ask God to take him soon. They should better help me with the treatment”, says the mother between tears.
He adds that the baby is neurally active, has gained weight and has had no complications. “If they put the medicine on him right now, he would recover, he would start moving his body. They are very unfair, they do not want to help us”.
Fernando has a job for which he receives the basic salary, but he also sells sheets to obtain more income and bear the costs of his son’s illness. In addition, the family has organized activities such as raffles or solidarity meals, but nothing is enough.
Theo and Maikel’s families have launched campaigns through social networks to obtain the two million dollars they need to buy Zolgensma, as well as to pay for additional expenses: travel, medicine, therapies, devices, etc.
Those who wish to collaborate with Theo can contact the mother at the following telephone number 0967595863. Or deposit to the Banco del Pichincha savings account № 2203527162 in the name of Laura Varela. ID number: 1003549183.
To collaborate with Maikel and his family, you can contact the number 0979498421. Or deposit to the savings account № 5976150200 of 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 conditions that damage and kill the motor neurons that control movements of the arms, legs, face, throat, and tongue.
As the neurons die, the muscles begin to weaken and atrophy. It is a progressive wear that affects speaking, walking, swallowing and breathing, as described by the portal MedlinePlus.
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 Ecuador Spinal Muscular Atrophy Foundation (FAME), there are 23 patients suffering from the disease in the country.
There are three treatments approved by the 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 Noguera, president of the Ecuador Spinal Muscular Atrophy Foundation (FAME) states that, although these drugs are not part of the National Medicines Table, “it is the Government’s obligation to provide medicines for this class of diseases.”
He adds that last year they had unsuccessful meetings with the then Minister of Health, Ximena Garzón, who promised to sign agreements to speed up the care of minors, but nothing ever materialized. “These are children who die daily, we are already with five losses last month.”
LIFE OR DEATH
Noguera also has a son with grade 2 SMA. She describes her struggle as terrible. At the beginning there was no life expectancy for the little one, much less treatment for him. Then came the joy that the FDA approved the drug.
Daniel is 12 years old, but he has not had access to the medicine for more than five years. The request has been repeatedly denied by the Baca Ortiz Hospital.
“Without medicine, the disease damages every function. What the government is doing is waiting for time to pass, for the children to degenerate and then saying that 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 peacefully, knowing that its people are dying for lack of medicine. As it is a rare disease, they have shamelessly told us that they prefer to save 30 children with cancer than one with spinal muscular atrophy”.
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Daniel, Elizabeth Noguera’s son, has type 2 SMA and has been struggling to obtain his medication for 5 years.
The foundation calls on the Minister of Health, José Ruales, to meet with the families and to sign agreements for the acquisition of medicines, as soon as possible.
Vistazo requested an interview with the Ministry of Health for a week, but until the closing of the note, he has not received a response.