Genes shape brain structure and may influence neurological disorders like Parkinson’s and ADHD, revealing new insights into brain health.

Sickle cell disease is a severe, inherited blood disorder that primarily affects people of African, Mediterranean, Middle Eastern, and Indian descent.Despite its widespread impact, SCD research has ...

The nonprofit foundation, n-Lorem developed personalized medicine for a child with rare leukodystrophy.

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

Tiny Michigan biotech startup CircNova has raised a $3.3 million seed round for its technology that uses AI to target ...

Despite the prevalence of cardiovascular disease, many people are unclear about the role their genes play and how to lower their risk.

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 ...

Mirum Pharmaceuticals' Ctexli wins FDA approval for CTX, a rare lipid storage disease. The company projects 2025 global sales of $420–$435 million.

A clinical research team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international genetic researchers led a global research study using multi-omics analysis and ...

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

It’s a race against a single genetic marker -- and musician turned biotech start-up CEO Casey McPherson says the stakes for ...

A 75-year-old man with a genetic predisposition for Alzheimer’s has yet to show signs of the disease. Could this offer ...