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Barth Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Barth Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Barth Syndrome trials you may qualify forThe purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired pri…
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disord…
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide particip…
Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily S…
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor to learn more about this program. The treating ph…