The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first ...

Researchers from the Mitochondrial Medicine Program at Children's Hospital of Philadelphia (CHOP) have better characterized a spectrum of what were classically considered discrete mitochondrial DNA ...

An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment started in the womb.

Dr. Steve Skinner, president and CEO of the Greenwood Genetic Center, was honored with the 2025 S.C. Life Sciences Pinnacle ...

A 75-year-old man with a genetic predisposition for Alzheimer’s has yet to show signs of the disease. Could this offer insights on new preventive and therapeutic avenues?

A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...

Explore new findings on bipolar disorder, from genetics to sleep patterns, plus emerging treatments and a neuroscientist’s ...

Epilepsy is a complex disorder with both obvious and hidden symptoms, and some individuals are at a higher risk due to ...

March 2 is Eating Disorder Awareness Week. Johns Hopkins University Hospital answers five frequently asked questions about eating disorders.

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...