PCR137 Within-Trial Interviews to Contextualize the Patient Experience and Supplement Clinical Outcomes Assessments in a Pivotal Trial of a Newly Described, Fatal and Ultra-Rare Pediatric Disease
Abstract
Authors
L Litcher-Kelly A Ozen S Ollis Baris Feldman H A Yaworsky P Medrano V Chongsrisawat T Brackin L Perlee M Walker S Pradeep MJ Lenardo O Harari JJ Jalbert