After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!
Tag Archive: mitochondrial disease
Certain defects in the way the mitochondria work may lead to diabetes, and the ways our bodies adapt to high blood sugars share many features with the ways our bodies respond to mitochondrial disease.
By using existing human drugs to improve metabolism and restore shortened lifespans in microscopic worms, scientists have set the stage for human clinical trials of possible innovative therapies for mitochondrial disease.
A new approach being developed at The Children’s Hospital of Philadelphia and the University of Pennsylvania could allow investigators to know within days if a chosen therapy reverses the intracellular mechanisms that go awry in Friedreich’s Ataxia (FA), a genetic mitochondrial disease
Children’s Hospital researchers recently identified a network of signaling molecules that acts like a “fuse box,” regulating the effects of defective energy flow in mitochondrial respiratory chain diseases.