Meet some of our patients

Rhys

Born with X-linked Severe Combined Immunodeficiency (X-SCID), a rare genetic condition affecting the immune system in boys, Rhys was unable to fight infection. Some children with X-SCID may have a bone marrow transplant if a donor can be found, but for Rhys this was not an option. His only chance was through pioneering gene therapy.

Rhys' bone marrow was removed from his body, mixed in a test tube with a normal copy of his faulty gene and returned to his body. Within four weeks, Rhys was showing signs of developing a fully-working immune system, and seven months later he needed no artificial immune system support at all.

Rhys is a miracle case, and according to his mother, Marie:

"We do all the things that other families do – go on holiday, play in the park. Things that would have been impossible before without the miracle work of GOSH."

Rhys was the first case to be treated at GOSH following the opening of a specialist laboratory to facilitate clinical trials of gene therapy.

Meet other patients here.