Mitochondrial Disease News and Research

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Research breakthrough opens door to a world of regenerative medicine for treating mitochondrial disease

A study led by Shoukhrat Mitalipov, Ph.D., and Hong Ma, M.D., Ph.D., at the Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University and the Oregon National Primate Research Center has revealed the first critical step in developing novel gene and stem cell therapy treatments for patients with mitochondrial disease.

16 Jul 2015

Dietary fat intake could potentially ease mitochondrial disease, shows research

Mice that have a genetic version of mitochondrial disease can easily be mistaken for much older animals by the time they are nine months old: they have thinning grey hair, osteoporosis, poor hearing, infertility, heart problems and have lost weight. Despite having this disease at birth, these mice have a "secret weapon" in their youth that staves off signs of aging for a time.

1 Jul 2015

NGS technology helps identify new mitochondrial disease gene

Next Generation Sequencing (NGS) technology offers an incredible opportunity for the rapid and relatively low-cost characterization of individual genomes, giving us a chance to make a substantial leap ahead in the molecular dissection of all mitochondrial disorders in humans.

27 Jun 2015

Novel findings may hold promise for children, adults with mitochondrial disorders

Rooted in malfunctions in the tiny power plants that energize our cells, mitochondrial disorders are notoriously complex and variable, with few effective treatments. Now, novel findings in microscopic worms may hold great promise for children and adults with mitochondrial disorders

16 Apr 2015

Study opens door for development of tools to modulate CoQ10 synthesis in human cells

A study which counts with the participation of University of Granada scientists has provided new data on the Q10 coenzyme (CoQ10), a molecule which is synthesized within the cells of the organism itself and which has essential functions for cellular metabolism.

4 Feb 2015

Penn State scientists find that mitochondrial disease more prevalent in older moms

The discovery of a "maternal age effect" by a team of Penn State scientists that could be used to predict the accumulation of mitochondrial DNA mutations in maternal egg cells -- and the transmission of these mutations to children -- could provide valuable insights for genetic counseling.

14 Oct 2014

UCLA research may lead to novel treatment for PH1

Research by UCLA biochemists may lead to a new treatment - or even a cure - for PH1, a rare and potentially deadly genetic kidney disease that afflicts children. Their findings also may provide important insights into treatments for Parkinson's disease, Alzheimer's disease and other degenerative diseases.

27 Sep 2014

ChromaDex to provide Murdoch Childrens Research Institute with NR ingredient for research use

ChromaDex Corp., an innovative natural products company that provides proprietary ingredients and science-based solutions to the dietary supplement, food and beverage, animal health, cosmetic and pharmaceutical industries, announced today it has entered into a material transfer agreement (MTA) with Murdoch Childrens Research Institute, the preeminent child health research institute in Australia.

31 Jul 2014

Exome sequencing improves diagnosis of mitochondrial disease

Use of exome sequencing improved the ability to identify the underlying gene mutations in patients with biochemically defined defects affecting multiple mitochondrial respiratory chain complexes (enzymes that are involved in basic energy production), according to a study in the July 2 issue of JAMA.

2 Jul 2014

FMM supports research project to investigate mitochondria-targeted therapeutic approach

The Foundation for Mitochondrial Medicine announced today that it is working with The Michael J. Fox Foundation for Parkinson's Research to support a research project led by Wolfdieter Springer, PhD, at the Department of Neuroscience at Mayo Clinic in Florida to investigate a mitochondria-targeted therapeutic approach to treating Parkinson's disease.

26 Jun 2014

Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome.

9 Jun 2014

Edison Pharmaceuticals' EPI-743 granted orphan designation for Leigh syndrome in Japan

Edison Pharmaceuticals has announced that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Designation to EPI-743 for the treatment of Leigh syndrome.

21 May 2014

Researchers examine link between alcoholism and loss of muscle strength

Muscle weakness is a common symptom of both long-time alcoholics and patients with mitochondrial disease. Now researchers have found a common link: mitochondria that are unable to self-repair.

22 Apr 2014

Neurovive presents new breakthrough on energy regulation at cellular level

NeuroVive, a leading mitochondrial medicine company, is presenting a breakthrough in the company's work on energy regulation at the cellular level.

17 Apr 2014

Research suggests that person can slow aging process by exercising regularly

New research by Canadian sports medicine physician Mark Tarnopolsky, MD, PhD, suggests that a person can slow the speed at which they age by exercising regularly. Dr. Tarnopolsky presented his research titled, "Exercise as a Countermeasure for Aging: From Mice to Humans" today at the 23rd Annual Meeting of the American Medical Society for Sports Medicine (AMSSM). Dr. Tarnopolsky discussed how regular exercise not only improves the quality of life but can also extend a person's lifespan by up to five years.

8 Apr 2014

Researchers show that vitamin B3 can slow down progression of mitochondrial disease

The researchers of the University of Helsinki, Finland, and école Polytechnique Fédérale de Lausanne, Switzerland, have shown that vitamin B3 form nicotinamide riboside can slow down the progression of mitochondrial disease, suggesting its potential as a novel therapy approach to adult-onset mitochondrial muscle diseases.

7 Apr 2014

Researchers find new drug target for mitochondrial dysfunction

Mitochondria, long known as "cellular power plants" for their generation of the key energy source adenosine triphosphate (ATP), are essential for proper cellular functions. Mitochondrial defects are often observed in a variety of diseases, including cancer, Alzheimer's disease, and Parkinson's disease, and are the hallmarks of a number of genetic mitochondrial disorders whose manifestations range from muscle weakness to organ failure. Despite a fairly strong understanding of the pathology of such genetic mitochondrial disorders, efforts to treat them have been largely ineffective.

28 Mar 2014

Edison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson syndrome

Edison Pharmaceuticals today announced the initiation of a phase 2 study entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone™) in Children with Pearson Syndrome." The Investigative New Drug application (IND) was approved by the Food and Drug Administration, Office of Hematology and Oncology Products.

17 Mar 2014

FDA grants Fast Track designation to Edison’s lead drug EPI-743

Edison Pharmaceuticals today announced that the United States Food and Drug Administration granted Fast Track designation to EPI-743, the company's lead drug, for the treatment of Friedreich's ataxia. EPI-743 is being developed for pediatric and adult mitochondrial disease, including Friedreich's ataxia.