Baby with neurodegenerative disease receives revolutionary therapy at Pequeno Príncipe Hospital

September 16, 2020 will be marked in the history of Pequeno Príncipe and in the life of ten-month-old baby Amanda Soave, born in Xanxerê, state of Santa Catarina. It was on that day that she received, at Pequeno Príncipe Hospital, Zolgensma, known as the most expensive medicine in the world, which a single dose costs about US$ 2 million. Amanda was the first Brazilian child to receive medication to treat Spinal Muscular Atrophy (SMA) free of charge through the global access program created by the pharmaceutical company Novartis, the product developer.

The Pequeno Príncipe was one of five Brazilian hospitals certified to apply the medication. “We were authorized to apply because we were accredited by the National Biosafety Commission, meeting standards for handling genetically modified organisms. In addition, we have a team specialized in the care of this disease, formed by neuropediatricians, physiotherapists, speech therapists and other professionals,” highlights the technical director of the Hospital, Donizetti Dimer Giamberardino Filho.

In the 14th edition of Pequeno Príncipe News – Special edition COVID-19, you will learn more about the story of little Amanda. You will also know more details about two articles published in September in Science magazine which show that more than 10% of young, healthy people who develop severe forms of COVID-19 produce antibodies that do not attack the virus that causes the disease, but the immune system itself (autoantibodies). Another 3.5%, at least, carry a specific type of genetic mutation that affects the immune response. The findings are part of the research conducted by “COVID Human Genetic Effort”, an ongoing international project that covers more than 50 genetic sequencing centers and hundreds of hospitals worldwide. In Brazil, the research is led by the scientist of the Pelé Pequeno Príncipe Research Institute, Carolina Prando, together with the researcher from the University of São Paulo (USP), Antonio Condino Neto.

“The findings explain why some people develop a much more serious disease than others in their age group – including, for example, individuals who needed to be admitted to the ICU despite being in their 20s and free from comorbidities,” explains the scientist, who will also be the honoree of Pequeno Príncipe Gala 2020 – A Journey of Hope, event held virtually this year due to the pandemic, on November 18.

COVID-19 Report
The Pequeno Príncipe News also presents a report about the patient care offered to boys and girls with suspected of COVID-19. Since March 4 until October 9, the Hospital investigated 822 patients with suspect of the disease and 120 cases have been confirmed. Of these, on October 9, 63 were sent to isolation at home, 52 were recovered and, unfortunately, five patients died.

Click here and read the full-length articles in the 14th edition of Pequeno Príncipe News – Special edition COVID-19.

Click here and learn more about the use of the funds raised in 2022 Gala