Leigh Syndrome News and Research

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Hypoxia therapy shows promise for treating mitochondrial brain disorders

Oxygen isn't always a good thing. Of course, people-and most organisms-cannot live without it. But oxygen can also be quite toxic and lead to profound health consequences.

8 Jul 2026

Viagra ingredient improves symptoms in patients with Leigh syndrome

Sildenafil – an active ingredient also marketed under the name of Viagra – improves symptoms in patients with Leigh syndrome.

11 Mar 2026

New discovery may aid in early detection of rare genetic disease

Virginia Tech researchers discovered an indication hidden in the brain that may help doctors identify children suffering from a rare genetic disease earlier. Their findings were published recently in EMBO Molecular Medicine.

28 Jan 2026

New mechanism behind potentially fatal type of epilepsy identified

A team led by the UAB Institut de Neurociències (INc-UAB) has for the first time identified the mechanism behind a potentially fatal type of epilepsy, a symptom of mitochondrial diseases.

27 Oct 2025

Low oxygen environment protects brain and restores movement in mice with Parkinson's

Researchers from the Broad Institute and Mass General Brigham have shown that a low-oxygen environment - similar to the thin air found at Mount Everest base camp - can protect the brain and restore movement in mice with Parkinson's-like disease.

6 Aug 2025

Mitochondria depletion in stem cells offers insights into cellular energy and evolution

By using a genetic technique developed at UT Southwestern Medical Center that forces cells to rid themselves of mitochondria, researchers are gaining new insights into the function of these critical organelles.

7 Jul 2025

New drug mimics low-oxygen treatment for Leigh Syndrome

For the average person, living at high altitude-where oxygen is sparser than at sea level-can have health benefits ranging from lower rates of heart disease to increased endurance.

18 Feb 2025

Cannabidiol shown to extend lifespan and improve symptoms in Leigh syndrome models

A study led by the UAB Institut de Neurociències and published in the journal Nature Communications demonstrates in animal models how daily administration of cannabidiol (CBD), a substance obtained from the cannabis plant, extends lifespan and improves symptoms associated with Leigh syndrome.

6 Sep 2024

Researchers uncover mechanism behind mitochondrial dysfunction in Alzheimer's

A team of researchers at Johannes Gutenberg University Mainz (JGU) has identified a mechanism that causes mitochondrial dysfunction in Alzheimer's patients resulting in a reduction of the supply of energy to the brain.

25 Apr 2024

Hope for PDCD and Cure Mito Foundation unveil joint patient registry

Cure Mito Foundation and Hope for PDCD foundation, both patient-led foundations focused on advancing research and supporting families affected by Leigh syndrome and Pyruvate Dehydrogenase Complex Deficiency (PDCD), respectively, are excited to announce a launch of a PDCD patient registry.

18 Mar 2024

CoRDS and Cure MITO join forces with C-Path to advance research for rare mitochondrial disorders

Critical Path Institute announced today that the Coordination of Rare Diseases based at Sanford Research (CoRDS), in partnership with Cure MITO, will contribute its mitochondrial disorders data from its international patient registry to the C-Path-managed Rare Disease Cures Accelerator-Data and Analytics Platform .

13 Jul 2023

Cure Mito Foundation releases the first-of-its-kind online resource about Leigh syndrome

The Cure Mito Foundation, a parent-led organization dedicated to advancing research and treatments for Leigh syndrome, has launched the first-of-its-kind online resource about Leigh syndrome, the most common type of pediatric mitochondrial disease.

5 Jun 2023

Research reveals molecular features of rare mtDNA mutations

A high-throughput single-cell single-mitochondrial genome sequencing technology known as iMiGseq has provided new insights into mutations of mitochondrial DNA (mtDNA) and offers a platform for assessing mtDNA editing strategies and genetic diagnosis of embryos prior to their implantation.

13 Apr 2023

Two new zebrafish models for studying the most common cause of Leigh syndrome developed

Researchers from Children's Hospital of Philadelphia (CHOP) developed two new zebrafish models for studying a specific genetic form of mitochondrial disease that represents the most common cause of Leigh syndrome.

21 Feb 2023

Parents become drug developers to save their children’s lives

Maggie Carmichael wasn't developing like other kids. As a toddler, she wasn't walking and had a limited vocabulary for her age.

21 Jul 2022

Study finds microglia to be responsible for neuronal death in a mitochondrial disease animal model

Microglia, a type of central nervous system cell, is primarily responsible for neuronal death in Leigh Syndrome and the neurological symptoms related to this mitochondrial disease.

8 Jul 2022

Developmental delays related to Leigh syndrome may occur earlier than previously known

Researchers from the Mitochondrial Medicine Frontier Program at Children's Hospital of Philadelphia have found that developmental delays associated with Leigh syndrome, the most common pediatric mitochondrial disorder, may occur earlier than previously recognized – even before metabolic stroke and regression – which could provide clinicians with an opportunity for earlier diagnosis and therapeutic interventions.

1 Mar 2022