A mother has chosen to return to her home country with her 11-year-old daughter, who has a rare genetic disorder, after being ...

Musician Casey McPherson gave up his career to find a cure for his 9-year-old daughter Rose, who has an ultra-rare genetic ...

Angelman Syndrome is a rare genetic condition that effects one in every 20,000 kids. Clinic at Children's Hospital Colorado ...

You’ve likely heard people mention that they have inherited certain health conditions from their family. These are known as genetic disorders—conditions passed down from parents through DNA. In this ...

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other ...

Marley Mansour, 11, is one of fewer than 100 people in the world with a particular mutation in the NARS1 gene. She's set to ...

A nonprofit called the n-Lorem Foundation is working on developing an individualized treatment that targets the genetic cause of rare diseases.

Researchers at UC Berkeley’s Innovative Genomics Institute, or IGI, developed a revolutionary treatment using CRISPR gene ...

Fargo’s Aldevron helped create a custom gene-editing therapy that cured a baby of urea cycle disorder, a rare and potentially ...

He’s among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...