Elania is one of 59 children born with ADA-SCID who have been cured using this gene therapy, which will soon be submitted for ...

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

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

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

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

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

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

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

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

In a note to investors on Sunday, analysts at Leerink Partners called these findings “compelling,” adding that they indicate ...