Hecad performs first gene therapy for Spinal Muscular Atrophy

The Hospital Estadual da Criança e do Adolescente (Hecad) has performed its first administration of the gene therapy medication Zolgensma to treat Spinal Muscular Atrophy (SMA) 5q. The patient, a six-month-old infant, received a single dose through the Unified Health System (SUS).

Zolgensma is a highly advanced treatment that addresses the genetic cause of SMA by providing a functional copy of the gene responsible for producing the protein necessary for motor neuron survival. The medication was provided by the Ministry of Health. Each dose is estimated to cost approximately R$ 10.457 million, making it one of the most expensive therapies available for rare diseases.

The procedure followed strict clinical evaluation and eligibility protocols established by the SUS. To prepare for the treatment, Hecad staff underwent specific training regarding the administration and the necessary care before, during, and after the infusion.