Barth Syndrome
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Ph3b/4 Trial in Patients With Barth Syndrome [Phase 4]
Notes a Phase 3b/4 clinical trial in Barth Syndrome, sponsored by Stealth BioTherapeutics, that is not yet recruiting.
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CLINICAL_TRIALS research · 2025-02-14Clinical Trial Update: An Intermediate Size Expanded Access Protocol of Elamipretide
The update describes an intermediate-size expanded access protocol for elamipretide in patients with mitochondrial diseases, including Barth…
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CLINICAL_TRIALS trial_result · 2024-04-16Clinical Trial Update: A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome [Phase 2, Phase 3]
Phase 2/3 trial evaluating safety, tolerability, and efficacy of elamipretide in Barth syndrome completed with results posted in 2024.
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CLINICAL_TRIALS trial_result · 2017-01-10Clinical Trial Update: Exercise Training in Barth Syndrome [NA]
This update announces the completion of a clinical trial evaluating exercise training in Barth syndrome.