An N-of-1 Trial in a Progressive Rare Disease: A Case Report on Design, Consent and Interpretation. Translational Medicine & Therapeutics Journal, [S. l.], v. 2, n. 1, p. 37–45, 2023. DOI: 10.5281/zenodo.22649050. Disponível em: https://inkwise.org/index.php/TMTJ/article/view/81. Acesso em: 5 nov. 2026.