A Brazilian child suffering from spinal muscular atrophy (SMA) who won in court the right to receive the most expensive drug in the world showed improvements after taking the drug, his mother, Jozelma Silva, reveals.
Jozelma told me G1 that her daughter, Júlia Maria, started moving her legs and being able to move after taking the medication. “She started showing improvements. My little girl wouldn’t move her legs before the medication,” he said.
The child needed the drug Zolgensma, which costs about six million reais (1.15 million euros). In September this year, the Federal Supreme Court ruled that Brazil’s healthcare system must provide the girl with the drug.
Júlia Maria was released from the hospital in Curitiba, where she was given medication, and will undergo a new evaluation on Friday. “If all goes well, she will return to Fortaleza on Saturday,” the mother said.
“Everyone knows the evolution of those who took Zolgensma and those who did not. We cannot believe it when we see it. I know that if Júlia takes it, she will walk. I hope Júlia will come off the ventilator that she will breathe independently. It will improve her quality of life in many ways,” the mother emphasizes.
Spinal muscular atrophy (SMA) is a rare, degenerative and hereditary disease that has no cure and disrupts the body’s ability to produce a protein essential for the survival of motor neurons, which are responsible for simple vital body movements , such as breathing, swallowing and movement. The disease ranges from type 0 (before birth) to type 4 (second or third decade of life).
According to the Brazilian Ministry of Health, Zolgensma is a single-dose intravenous drug and positively affects breathing, chewing, tongue movements, swallowing, gag reflex and speech articulation in people with type 1 SMA.
Source: DN
