Doenças Raras na Infância: Avanço no Tratamento da Fibrose Cística com Vanzacaftor–Tezacaftor–Deutivacaftor aprovado pela FDA para Crianças

Referências

1. Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, et al. Vanzacaftor–tezacaftor–deutivacaftor for children aged 6–11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025;[publicado online]. Disponível em: https://doi.org/10.1016/S2213-2600(24)00407-7.

2. Vertex Pharmaceuticals. Vertex announces U.S. FDA approval of ALYFTRKETM, a once-daily next-generation CFTR modulator [Internet]. Disponível em: https://investors.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-alyftrektm-once-daily-next