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Congenital athymia is an ultra-rare pediatric condition characterized by the lack of thymus in utero and the naïve T cells critical for infection defense and immune regulation.
Rodatristat ethyl is a first in class prodrug for rodatristat, a potent inhibitor of tryptophan hydroxylase, the rate limiting enzyme in the biosynthesis of serotonin from dietary tryptophan.
This cross-sectional study of adult caregivers of patients with congenital athymia used both a quantitative survey and qualitative interviews. Caregivers of patients currently receiving supportive care responded to questions about the past 12 months.