Mitochondria genetics may be more complex than previously thought, with new research reporting that DNA content can vary significantly among different mitochondria within a single cell, and between mice and people — a finding of potential importance to research that relies on mouse models of disease. The work may also…

NeuroVive Pharmaceutical has made significant advances in its NVP015 program for mitochondrial diseases with the identification of a lead compound to develop in preclinical studies. Preliminary analyses in experimental models of genetic mitochondrial disease revealed one specific compound that was well tolerated and able to reach the brain, while…

KH176 by Khondrion had a favorable safety profile and improved depression and migraine in some forms of mitochondrial disease, according to preliminary results from an ongoing Phase 2 trial. The study included carriers of a gene defect called m.3243A>G who have MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) and…

Researchers found that patients with different types of mitochondrial disease have higher rates of headaches compared to the general population. Tension-type headaches (TTH) were more frequent than migraines among this patient population, the study found. The study, “Prevalence of Headache in Patients With Mitochondrial Disease: A Cross-Sectional Study,” was…

NeuroVive Pharmaceutical has received a signal from European regulators that its mitochondrial disease therapy KL1333 is likely to receive Orphan Drug Designation, which could accelerate its development and approval. The company is seeking Orphan Drug Designation for KL1333 as treatment for a condition known as mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like…

Khondrion has secured three new patents that protect several products, including the Dutch pharmaceutical company’s lead candidate, KH176, to treat mitochondrial disease and related conditions. KH176 is a small molecule that targets reactive oxygen species, or byproducts of mitochondria. The compound belongs to a new class of drugs that…

Mitochondrial disorders often affect several organs, but symptoms are more likely to be evident in organs like the heart that are most dependent on oxidative metabolism — the use of oxygen to convert sugars to energy. But while the diagnosis and treatment of cardiac problems related to mitochondrial disease is advancing, it still falls…

Reduced exercise capacity in people with mitochondrial diseases is linked to both a heart that works inefficiently and muscles that inadequately extract oxygen, according to a study that pinned down the physiological contributions that limit a patient’s ability to perform physical activity. These mechanisms differ between patients with mitochondrial diseases and those…

Korean researchers have enrolled the first participant in a Phase 1 clinical trial of KL1333 as a treatment for genetic mitochondrial diseases such as MELAS and Kearns-Sayre syndrome. The healthy participant will be part of a study evaluating the therapy’s safety, tolerability, stability and the way it distributes itself in…

A mitochondria-targeting compound called mitochonic acid (MA-5) had beneficial effects on mitochondrial function, energy production, and the formation of oxidative molecules in experiments involving a mouse model of mitochondrial disease and cells from patients. The study also identified a biomarker of mitochondrial disease and the response to MA-5 — a…