Survival and Clinical Progression in Barth Syndrome: Insights From the Barth Syndrome Foundation's Database of 502 Affected Individuals.

J Inherit Metab Dis · 2026 · PMC13136057 · PMID 42070974

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showed a trendno p-value reported

Evaluation of clinical manifestations over time showed a trend toward increased frequency of manifestation across all categories evaluated, further underscoring that BTHS is a progressive disease and not one that gets better if the affected individual survives past age 3.

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