The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare ...

This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which ... a fatal disease in children that could be prevented with the advances in gene therapy.

KJ Muldoon came into the world with a genetic time bomb ticking inside him.

Mansour is set to make medical history as the first person in the world to receive treatment for NARS1, a rare genetic disorder. (Juan Figueroa / Staff Photographer) An 11-year-old from Rowlett is ...

A baby in the US is among the first people with a rare genetic disorder to be treated with CRISPR, a customised gene-editing therapy that allows scientists to edit DNA. The baby, known as KJ ...

BALTIMORE, MD – A newly identified genetic disorder causing rapid aging has been named Garg-Mishra Progeroid Syndrome (GMPGS) in honor of its discoverers, Dr. Abhimanyu Garg and Dr. Prashant Mishra.

Ocugen ( NASDAQ: OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric ...

The Pennsylvania baby was born with a disorder that causes dangerous ammonia levels to build in the blood. China fires warning at US treaty ally 11 Expensive Steakhouse Chains That Aren't Worth ...