When large quantities are accumulated lactate can be damaging. An unblinded experimental treatment protocol of dichloroacetate treatment has been underway for 2 years. Results are encouraging but it has become apparent that a larger study will be needed to answer the questions which have arisen about efficacy and risks of this treatment. We hope to be able to identify which patients will benefit from dichloroacetate treatment and which patients will not. Neuroimaging in Leigh's Syndrome Leigh's syndrome is a devastating childhood form of mitochondrial disease.
Diagnosis is often difficult and neuroimaging studies are often used to help.
In our study of over 20 patients with Leigh's syndrome we have found previously unreported patterns of disease on magnetic resonance brain images and in some patients dramatic changes over time. This research will improve the clinical diagnosis of Leigh's syndrome.