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

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

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

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using ‘longevity genes’ ...

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

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

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

Through the newly-formed Singapore-Shanghai Medical Innovation Centre, clinicians from both hospitals partner to advance breakthroughs in cell and gene therapy and orthopaedics for a start ...

Researchers convert type A kidney to type O, potentially reducing wait times and saving lives. Experimental gene therapy ...

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

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

Stevie Ringel is the founder of the Kizuna Foundation, an organization seeking a treatment for a genetic retinal mutation ...