Children who need treatment of Type 1 Spinal Muscular Atrophy (AME), rare disease that affects body movements and also breathing, will have, from Monday (24), access to innovative and free treatment by the Unified Health System.
“It is the first gene therapy that is being introduced into the Unified Health System. SUS becomes part of a small club of five national public systems worldwide to offer this medicine to its population,” said Health Minister Alexandre Padilha.
The indication of this type of therapy is for patients up to 6 months of age that do not have invasive mechanical ventilation over 16 hours a day. According to the Ministry of Health, with the incorporation of Zolgensma, the SUS will offer to love type 1 all the therapies of this disease.
According to data from the Brazilian Institute of Geography and Statistics (IBGE), among the 2.8 million Brazilians living in 2023, about 287 were diagnosed with the disease. In practice, treatment makes use of the drug to replace the function of an absent gene or not working properly.
The incorporation of treatment was made possible through an agreement signed with the international industry, which conditions the payment to the result of therapy in the patient.
“It will permanently follow the assessment of the performance of the drug, the improvement of the life of this child and the family life, with clinical evaluation markers, throughout the treatment,” explains the minister.
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Protocol
To start treatment, the patient’s family should look for one of the 28 reference services for gene therapy of AME, present in the states of Alagoas, Bahia, Ceará, Federal District, Espírito Santo, Goiás, Minas Gerais, Mato Grosso, Pará, Paraíba, Pernambuco, Piaui, Paraná, Rio Grande do Norte, Rio Grande do Sul, Santa Catarina and Sao Paulo.
The patient will be welcomed and will undergo a screen -oriented screening and therapeutic guidelines for spinal muscle atrophy 5q types 1 and 2, established by the Ministry of Health. In the coming weeks, a committee will also be installed for permanent monitoring of patients who will use the single dose medicine.
Prior to the agreement, Zolgensma was already offered by the Ministry of Health in compliance with 161 lawsuits.
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Understand
AME is a rare disease that interferes with the ability to produce an essential protein for the survival of motor neurons, responsible for simple vital volunteer gestures, such as breathing, swallowing and moving. The disease is considered rare because it reaches less than 65 people in every 100,000 individuals.
In the public network, patients with types 1 and 2, in other age groups, are treated with drugs for continuous nusinersen and risdiplam. According to the Ministry of Health, in 2024, these drugs had 800 more prescriptions issued for treatment.
