Division of Human Genetics News

As clinical trials aim to attain the first FDA-approved treatments for mitochondrial disease, a study reports patient and family preferences.

Three Frontier Programs (NeoCLD Program, Mitochondrial Medicine, and Pediatric Airway Disorders) have made world-changing discoveries, delivered lifesaving therapies, and provided exceptional care.

Systematic laboratory studies find some antioxidants should be tested in clinical trials as treatments for patients with mitochondrial disease.

Children with mitochondrial diseases who suffered acute metabolic strokes benefited from rapid IV treatment with the amino acid arginine, with no side effects.

Lacking effective drug treatments, patients with mitochondrial disease may use many vitamins and supplements — most of which are unregulated and untested.

A breakthrough therapy has its genesis in more than 10 years of research conducted at Children's Hospital of Philadelphia.

A team of gene therapy researchers has reported on a highly active gene variant that achieved sustained factor levels in 10 adults, with no adverse effects.

Physicians who see patients with mitochondrial disease have a practical new tool: the first published care standards to guide clinical care.

Mitochondria, your cells’ power engines, could pay a key role in the development of autism spectrum disorder.

CHOP’s Douglas Wallace, a world-renowned pioneer in mitochondrial biology, received the 2017 Benjamin Franklin Medal in Life Science at the Franklin Institute.