Four-year-old Júlia Ortega Gonzalez Fernandes was given Zolgensma, a treatment that costs around $1 million, to improve her quality of life after being diagnosed with Spinal Muscular Atrophy (SMA) type 2.
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On National SMA Day, celebrated last August 8, her mother, Bruna Ortega Gonzalez Fernandes, expressed relief upon seeing small improvements in her daughter’s health.
Júlia, who lives in Bertioga, São Paulo, was diagnosed with SMA when she was almost two years old. The rare disease affects the ability to walk, run and even breathe. Since receiving Zolgensma in February, thanks to her health insurance, she has shown progress, albeit gradual.
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Before Zolgensma, Júlia took seven doses of Spinraza and the family had to go to court to obtain the most effective treatment. Now, the parents are struggling to get home care from specialized physiotherapists, which is essential for her continued development.
The girl’s mother, Bruna Ortega Gonzalez Fernandes, said that, little by little, her daughter has shown “small progress”. She also said she believes that the physiotherapy sessions will help her little one even more on this journey.
“We are still in this battle”, said Bruna. “We have to work on her hips, the thigh muscles and the positioning of her feet. She also has a bit of scoliosis, so [for] all of that we need physiotherapy”.
SMA is a rare genetic disease that interferes with the production of a protein vital for motor neurons, impacting essential functions such as breathing and movement. Zolgensma, a gene therapy, FDA-approved and is administered in a single dose, showing efficacy in combating the disease.
