News

July 13, 2017July 14, 2017

Charlie Gard Case Captures Vast and Unmet Needs of People with Mitochondrial Disease, Groups Say

The case of Charlie Gard — a baby in the U.K. with a terminal mitochondrial disease who ended up in a legal feud over continuing life support — illustrates ... Read more

July 12, 2017

Compound Called MITO-PIP May Have Potential as Gene Therapy for Mitochondrial Diseases

News

A compound that can silence a gene in a very specific manner was adapted by researchers to be used inside mitochondria — opening the possibility of manipulating genes linked to mitochondrial ... Read more

July 7, 2017July 7, 2017

First Participant Enrolled in Phase 1 Clinical Trial of KL1333 for Mitochondria Diseases

News

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 ... Read more

July 5, 2017July 5, 2017

Mitochondrial Disease an Enormous Burden on U.S. Patients, Study Finds

News

Mitochondrial disease is very costly to American patients and better therapies are needed urgently, say researchers at the Children’s Hospital of Philadelphia (CHOP). Their National Institutes of Health-funded study, “Hospitalizations ... Read more

June 30, 2017

Elamipretide Shows Therapeutic Potential for Primary Mitochondrial Myopathy in Latest Trial Results

News

The investigational drug elamipretide has therapeutic potential to improve the physical capacity of patients with primary mitochondrial myopathy (PMM), according to the latest results of the Phase 2 MMPOWER-2 study. ... Read more

June 23, 2017July 10, 2017

Mitochondrial Genetics Pioneer Douglas Wallace Wins Prestigious Research Award

News

Douglas Wallace, Ph.D., of the Children’s Hospital of Philadelphia, won the 2017 Dr. Paul Janssen Award for Biomedical Research for his pioneering work in the field of mitochondrial genetics. Founder ... Read more

June 16, 2017

GS010 Improves Vision in Patients with Mitochondrial Disease That Impacted Their Sight, Trial Shows

News

GenSight Biologics’ GS010 gene therapy restored some of the vision in Leber’s Hereditary Optic Neuropathy (LHON) patients whose sight had deteriorated, according to a Phase 1/2 clinical trial. The findings ... Read more

June 14, 2017

Increased Urea Levels Trigger Inflammation and Mitochondrial Dysfunction, Study Finds

News

Uremia (increased blood urea levels) may promote inflammation in the adipose tissue (fat) and faulty mitochondrial activity in the muscle, which can contribute to the development of end-stage renal disease, ... Read more

June 12, 2017June 12, 2017

Multiple Sclerosis Therapy Tecfidera Could Also Help Mitochondrial Disease, Friedreich’s Ataxia Patients, Study Finds

Mitochondrial disease, News

Tecfidera (dimethyl fumarate), an oral drug used to treat multiple sclerosis (MS), could also benefit mitochondrial disease patients as well as those with Friedreich’s ataxia (FA), concludes two studies from the ... Read more

June 9, 2017

Specific Compound Boosts Energy Output in Mitochondrial Disease Cells, Study Finds

News

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 ... Read more