Zolgensma gene therapy provides lifesaving option for Brazilian SMA patients

Zolgensma is a gene‑therapy medication that delivers a functional copy of the SMN1 gene, addressing the root cause of spinal muscular atrophy (SMA) type 1. It is administered as a single intravenous infusion and, in Brazil, is offered free of charge through the public health system (SUS) to infants up to six months old who are not on prolonged invasive ventilation.

The treatment costs roughly R$ 8.6 million per patient. A campaign in Santa Clara do Sul, Rio Grande do Sul, has mobilised volunteers, artists and local businesses to raise funds for one‑year‑old Léo Wolschick Soares, who needs Zolgensma before turning two. With a deadline of 22 August and about 35 days remaining, the effort has collected roughly R$ 1.2 million, selling hundreds of meals at a benefit concert and accepting donations via Pix and the Vakinha platform.