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

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Journal

Value in Health

Topics

Internal Medicine

Pediatrics

Medicine

Internal Medicine

Pediatrics

Genetics

Rare Disease

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DOI

10.1016/j.jval.2024.03.2016