Gene Therapy

Healthcare

Doctors say gene therapy slows Huntington’s disease for first time

It is the first time a drug trial has slowed the rare disorder’s progression in an ongoing, significant way, researchers said.

25 September 2025

Health news

Baby with rare disorder treated with personalised gene-editing therapy

The child will need to be monitored throughout his life, but researchers said the treatment’s success indicates that other patients could someday benefit from the approach.

16 May 2025

Health news

NHS to get access to gene editing therapy for sickle cell disease

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS.

31 January 2025

Healthcare

Draghi report touts path to pull EU pharma from ‘slow agony’

Mario Draghi envisions a clear path forward for the EU pharma sector through greater access to health data for AI development and targeted public investment in gene therapies, which he believes will help preserve the EU’s healthcare strengths.

10 September 2024

Health news

€2.5 million drug now approved in Ireland, Belgium and the Netherlands

Libmeldy - a treatment for a rare disease costing €2.5 million - has become available in Ireland, Belgium and the Netherlands.

26 January 2024

Health news

Experimental gene therapy allows kids with inherited deafness to hear

Gene therapy has allowed several children born with inherited deafness to hear.

25 January 2024