While Covid has made many aspects of living with a rare disease more difficult, it has inspired policies and techniques that could help rare disease research progress more smoothly both […]

Study is first to demonstrate how one specific combinational therapy may provide measurable benefits to patients with mitochondrial respiratory chain disorders, which currently lack FDA-approved therapies CHILDREN’S HOSPITAL OF PHILADELPHIA […]

What is pediatric mitochondrial disease? How does the COVID-19 pandemic affect children with this disease? Click here to read the full article written by Dr. Peter McGuire at the NIH […]

Early research shows how mild defects in the ‘batteries’ of cells in brain tissue can lead to behaviors similar to those of autism. Researchers at Children’s Hospital of Philadelphia (CHOP) […]

Graphic overview of the research. Credit: University of Granada Coenzyme Q10 (CoQ10) is a molecule essential for life that is synthesized in the cells of our organs and tissues, but […]