Fishman J, Anderson S, Talbird SE, Dingli D. Analysis of costs per responder in US adults with a paroxysmal nocturnal hemoglobinuria with suboptimal response to prior eculizumab treatment. Hematol Rep. 2023 Oct 13;15(4):578-91. doi: 10.3390/hematolrep15040060
Quittner AL, Sweeny S, Watrous M, Munzenberger P, Bearss K, Gibson NA, Fisher LA, Henry B. Translation and linguistic validation of a disease-specific quality of life measure for cystic fibrosis. J Pediatr Psychol. 2000 Sep 1;25(6):403-14.