Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

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

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

Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.

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

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 ...

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

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

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

These results are what we hoped for when we first began developing this approach,' one researcher said. View on euronews ...

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