Brazilian families seek funding for rare disease treatments

Families in Brazil are launching fundraising campaigns to secure expensive medical treatments for children suffering from rare conditions.

In Rio Brilhante, 8-year-old Miguel Baltazar is undergoing a medical struggle following a diagnosis of congenital megacolon. Since birth, Miguel has undergone ten surgeries to manage intestinal complications and attempts to reverse his colostomy. Following a recent attempt at reversal, a fistula developed, leading to an infection that required medical intervention and carries the risk of him needing to return to using a colostomy bag.

In Espírito Santo, the family of 5-year-old Gabriel is seeking to raise approximately R$ 17 million to fund a medical evaluation in the United States. Gabriel has been diagnosed with Duchenne Muscular Dystrophy (DMD), a progressive genetic neuromuscular disease. The family is aiming to access potential gene therapy to address the condition, which causes muscle tissue to be replaced by fat and fibrous tissue, eventually leading to mobility, cardiac, and respiratory complications.