Short-term treatment with oral KH176 was safe and well-tolerated by mitochondria disease patients, and lessened their symptoms of depression while also having a positive effect in alertness and mood, a Phase 2 clinical trial shows. The study with those findings, “The KHENERGY study: Safety and efficacy of KH176 in…

Analysis of a cerebrospinal fluid protein called neurofilament light (NfL), which is linked to nerve cell damage, can help monitor the neurodegenerative process in patients with mitochondrial diseases. This finding resulted from the study “Cerebrospinal fluid neurofilament light is associated with survival in mitochondrial disease patients,” which…

Khondrion’s mitochondrial disease treatment candidate KH176 has potential to be effective in a broad range of disorders, a new study reports. The research, “KH176 Safeguards Mitochondrial Diseased Cells from Redox Stress-Induced Cell Death by Interacting with the Thioredoxin System/Peroxiredoxin Enzyme Machinery,“ was published in the journal…

Investigational compound KL1333 was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of inherited mitochondrial respiratory chain diseases, NeuroVive Pharmaceutical AB recently announced. The designation is a promising step for NueroVive’s KL1333 program because it will provide extra support from the FDA, including regulatory and…

Errors in the chemical processing that takes place in the production of mitochondrial proteins may play a role in the development of certain mitochondrial diseases, scientists from Japan have found. The study, “Defective Mitochondrial tRNA Taurine Modification Activates Global Proteostress and Leads to Mitochondrial Disease,” was published in…

Side alternating vibration training (SAVT), which uses a vibrating platform to simulate exercise, can improve muscle power in patients with mitochondrial diseases, according to a study published in Archives of Physiotherapy. Mitochondrial diseases comprise a group of inherited disorders that changes the way cells in the body produce energy.

Children affected by mitochondrial diseases who show nutritional deficiencies improve gastrointestinal symptoms, quality of life, and catch up on their development after receiving nutritional support from a feeding tube, according to researchers. The study, “Enteral Tube Feeding in Pediatric Mitochondrial Diseases,” was published in the journal Scientific…

NeuroVive Pharmaceutical recently reported promising progress of its Phase 1 clinical trial evaluating KL1333 as an investigational treatment for genetic mitochondrial diseases. The trial is being led in collaboration with Yungjin Pharm in Korea, and explores KL1333 as a treatment for a series of…