First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of ...

Five out of six children with a type of hereditary hearing loss have shown improvement in their hearing and speech recognition after treatment with an experimental gene therapy. This is the first gene ...

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

NICE recommends life-changing gene therapy for children with ultra-rare genetic disorder The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...

The US Food and Drug Administration approved the first gene therapy to treat a rare and devastating muscle disease, but limited the approval to kids ages four and five based on currently available ...

Tom, 15, and Rosie, 10, carry a mutation of the DHDDS gene, a rare neurodegenerative condition.

'It could change lives': Inside London lab testing gene therapy for cancer and obesity Meira GTx, in Shoreditch, is working on potentially revolutionary treatments for a wide array of illnesses ...

Groundbreaking one-off gene therapy approved for severe sickle cell disease People in England with severe sickle cell disease will be among the first to receive treatment using revolutionary CRISPR ...

The EMA's human medicines advisory committee has recommended that BioMarin Pharmaceutical's haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to launch ...