Tests begin to apply the most expensive medicine in the world in SUS

A battery of exams will be performed on each patient, so that the possibility of using the medicine is attested. From there, the zolgensma dose will be applied. Unlike other treatments, the drug has a unique dose. It is suitable for children up to 6 months of age who are not in invasive mechanical ventilation over 16 hours a day.

AME is a genetic disease that has no cure. It interferes with the body’s ability to produce a special protein that guarantees the survival of the neurons responsible for motor activities. The muscles lose their strength, become increasingly weak and also lose muscle mass. The mutation can be inherited from one or both parents, or may occur spontaneously.

Remedy is considered an innovative therapy for the treatment of children. It is in the form of injection, applied to the vein. A gene that was not produced by the body for some reason is introduced into an inactive virus. When the immune system recognizes the presence of the “strange”, attacks the virus and releases the gene, which goes to the neurons. When it is installed, it can cause the proteins necessary so that the brain can understand that those activities exist.

Disease is the largest genetic cause of death in babies and children. Although more common, type 1 is the most severe and lethal. The diagnosis is divided into five subtypes, but unlike other diseases, as “smaller” the degree, the greater the impact on the patient’s life.

Existing therapies tend to stabilize the progression of the disease. According to the ministry, only six countries have zolgensma in the public network. Before SUS offers technologies to type I, children with the disease had a high probability of death before the age of 2. It is expected that, after the drug, children may have, even gradually, the ability to sit alone, swallow or chew.

For patients outside the aged range of Zolgensma, SUS guarantees two free drugs in the public network for types 1 and 2 of AME: nusinersena and Risdiplam. Only in 2024, more than 800 prescriptions of these medications were dismissed. Both are continuous use treatments.