New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...

“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...

On her mother’s instruction, a toddler gives her pink stuffed horse, Snap Snap, a big cuddle. Then, as told, she plants a ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder, a promising ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...