Treatment Trials

3 Clinical Trials for Various Conditions

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TERMINATED
Phase 2 Study of EPI-743 in Children With Pearson Syndrome
Description

Treatment of Pediatric Subjects with Pearson syndrome

RECRUITING
SLSMDS Natural History Study
Description

The Single Large-Scale mtDNA Deletion Sydrome: Natural History Study (PS-NHS) aims to collect data on standardized clinical outcomes, store data on the Champ Foundation Registry (CFR) and make this data available to researchers, clinicians, and industry partners who are studying SLSMDS to answer questions regarding the disease, including its causes, potential treatments, and other topics. A secondary aim is to analyze the data to understand research questions relating to the natural history of SLSMDS.

RECRUITING
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Description

The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.