Child with rare disease in AL has not yet received the most expensive medicine in the world from the SUS: ‘I don’t want to lose my daughter’, says mother | Alagoas

Maria Helena, aged 1 year and 11 months, cannot walk because of type 2 SMA

As the medicine is only recommended for children under 2 years of age and Maria Helena will turn 2 on April 3, the family is fighting against time to ensure that she receives the medication on time.

“The Brazilian leaflet says that it is only until the age of 2, because her immune system is ready and after that, the virus is very strong and her immune system will fight and even death can happen. That’s why it’s so It’s important to be until the age of 2. No one will be responsible for what happens after the age of 2, and I don’t want to lose my daughter”, said Dayze Kelly, Maria Helena’s mother.

Zolgensma is a type of medicine called gene therapy that returns the missing gene to the patient. The treatment is carried out in a single dose, with mandatory application in a hospital. Medication and hospital costs reach R$7.6 million.

The deadline for the Union to supply the medicine via SUS was five days. To g1, the Ministry of Health said that the demand was received by the Department of Demand Management in Judicialization in Health (DJUD/SE/MS), on March 18, and that the department adopted all measures to comply with the court decision , but did not provide a date for purchasing the medication.

Maria Helena’s family also appealed to the Government of Alagoas, which committed, through the State Department of Health (Sesau) to pay for the treatment at the hospital where the medication will be administered and travel.

“We sat down with Helena’s family. We had the news that the federal judge released the injunction for the purchase of the medication with the condition that the entire cost of the treatment, adjuvant treatments, travel and everything necessary for the application of the medication was paid for by the State”, said Health Secretary Gustavo Pontes in a video released by Sesau.

“We are responsible for all the costs of the treatment that will be carried out for her, which are treatments other than medication”, stated the secretary.

“The Union has started the purchase process. It will be taken at Hospital Santa Joana, in Recife, that has already been confirmed. We know which hospital will be applied, but we don’t know what day it will be, because it could be at any time “, said Maria Helena’s mother.

• SMA is a rare, degenerative disease, passed from parents to children and which has no cure.
• It is caused by changes in the gene that produces the SMN protein – which protects motor neurons;
• Without this protein, these neurons die and do not send nerve impulses from the spine to the muscles;
• This interferes with the ability to move, walk, swallow and even breathe. It can lead to death.
• The disease varies from type 0 (before birth) to 4 (second or third decade of life), depending on the degree of muscle impairment and the age at which the first symptoms appear;
• SMA affects 1 in every 11 thousand live births.

• The medicine is a gene therapy that returns the missing gene to the patient. It was developed by a laboratory in the United States, is sold by a Swiss company and costs around R$6 million. The National Health Surveillance Agency (Anvisa) approved the treatment in 2020.
• The gene is injected into the patient’s body through a virus capsule modified in the laboratory: they are the ones that transport the gene that the patient needs to the cells of the spinal cord;
• The smaller the child, the lower the weight and, therefore, the lower the amount of virus injected;
• Zolgensma is the only treatment available that acts directly on DNA;
• The application is made in a single dose;
• There is no change in the patient’s DNA;
• Clinical studies have already proven the effectiveness of the drug against SMA.