A child with SMA wins the right to take Zolgensma, the most expensive drug in the world

A three-year-old child from Espírito Santo, diagnosed with spinal muscular atrophy (SMA) won Justice the right to receive a single dose of Zolgensma, the most expensive drug in the world. Each dose of the drug, which is able to interrupt the disease progressioncosts about 10 million reais.

Zolgensma is applied in a single dose and, according to neurology experts, it is almost 100% effective. Until now, the capixaba child performed a alternative treatmentwhich consisted of applying Spinraza every four months.


This drug is also effective, however, it is more invasive, has many side effects and is used periodically. Zolgensma, in turn, corrects the genetic effect of SMA and allows the production of the protein that is blocked by the genetic defect causing SMA.

What is AME?

The soul is a degenerative disease which affects the body’s ability to produce survival motor neuron protein, or SMN protein. These neurons are responsible for essential voluntary movements, such as breathing, swallowing and moving.

The disease can manifest at different times in life, ranging from type 0, which appears even before birth, to type 4, in which the first symptoms appear in the second or third decade of the person’s life. SMA is the leading cause of infant mortality due to genetic diseases worldwide.

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In the case of Espírito Santo’s child, the first symptoms appeared when he was only four months old, which characterizes SMA type 1. His parents were already fighting in court for the right to treatment by Zolgensma, fully funded by the child. SUS since August 2020.

The case reached the Supreme Court

Plate indicating the Federal Court
The case was to be decided by the Supreme Court. Credit: Shutterstock

The case had been pending in court for about a year and a half and was not decided until the end of January, when the Federal Court of Justice (STF) authorized the application of the drug. The decision was complied with by the Union in early March and currently the drug is being imported.

The child is expected to receive the medicine this Friday (18), in a private hospital in Vitória, capital of Espírito Santo. The application takes about an hour and, if all goes well, the child will be released the following Saturday morning (17).

Via: victory sheet

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