“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 ...

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the ...

Study results published in The New England Journal of Medicine show the therapy significantly improved hearing in nearly a ...

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...

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

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

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

Abeona Therapeutics Inc. (Nasdaq: ABEO), a commercial-stage biopharmaceutical company developing cell and gene therapies for ...

Gene therapies hold immense promise for treating sight loss. These therapies use a modified, harmless virus to deliver therapeutic genes directly to diseased cells, helping them to function normally ...