Precision Medicine


Nina's story 

Read Nina's story, as told by her mum, Aga.


When Nina was born, everything seemed fine, but after a period of time we noticed she was not feeding properly and was sleeping more than usual. We started to become concerned and doctors soon discovered she had developed multiple infections. Soon afterwards she was placed in isolation to stop her picking up infections. We had to scrub down and wear masks and gowns before we could see her. Doctors confirmed she had a life threatening immune disorder and we were told that Nina would need a bone marrow transplant.

As Nina's health deteriorated she was moved to Great Ormond Street Hospital (GOSH). "Within weeks of being transferred doctors diagnosed her with ADA-SCID. Looking back, this was a turning point. Nina started to receive vital treatment to replace a key enzyme her body was not producing. She started to put on weight and the improvements she was making offered the possibility of gene therapy as a treatment."

Professor Bobby Gaspar, an expert in paediatrics and immunology at GOSH and the UCL Great Ormond Street Institute of Child Health, "discussed this option as an alternative to a bone marrow transplant and earlier this year she was given the treatment. Since the treatment, Nina's health has shown encouraging signs. However, we're still very cautious around her. We continue to have a strict regime of cleaning hands, surfaces and even sterilising toys to stop her coming into contact with infection as her immune system starts to build up. "The doctors and nurses at GOSH have been amazing. They're always available to answer all our questions and their approach to treating Nina has been extremely reassuring. This has given us the hope that one day Nina will be able to lead a good quality of life."

The UCL Great Ormond Street Institute of Child Health (ICH), together with its clinical partner, Great Ormond Street Hospital (GOSH) forms the largest concentration of children's research in Europe. 

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