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

L. Litcher-Kelly, A. Ozen,S. Ollis,Feldman H. Baris,A. Yaworsky, P. Medrano,V Chongsrisawat, T. Brackin, L. Perlee,M. Walker, S. Pradeep, M. J. Lenardo,O. Harari, J. J. Jalbert

Value in Health（2024）

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