Child with Rare 'Bubble Baby' Syndrome Trials World's First Cure
COMM: She may look like a normal healthy baby, but Nina Warnell suffers from an extremely rare condition that has left her without an immune system, meaning just a common cold could kill her. Graeme: Nina was born in Poland, which was where I've been working on a sercommon. Everything was good, the birth went well and we had a very healthy 9 pound daughter. Everything seemed fine, breastfeeding was going ok, then slowly over a period of time, we noticed less and less feeding and more and more sleeping, which was when we started to get concerned.
COMM: Nina was diagnosed with Severe Combined Immunodeficiency Syndrome. Known as 'bubble baby' syndrome, the rare condition effects just 1 in 300,000 babies. Aga: It was the hardest time of my life and I didn't know what was wrong with my baby and there was no one to give me those answers.
Graeme: You could just see her getting weaker and weaker and literally wasting away in front of your eyes. COMM: As Nina's health deteriorated, she was moved to Great Almond Street hospital. Doctors there saved her life and created a temporary immune system for her. Nina is now administered weekly enzyme replacement injections and given 15 medications each day. Graeme, Aga and their daughter Mia follow a strict daily regime of cleaning hands, surfaces, even sterilising toys.
Graeme: Our lives aren't the same as they used to be and it's a strange experience because you lose concept of what normal actually is. We don't really go out, we don't really socialise. My wife very much lives within these walls here, apart from the occasional times when we can, you know, go out in the push chair as long as it's fully enclosed in plastic sheeting, so we've lost a lot of social interaction/ COMM: Nina is now undergoing a cutting edge and pioneering form of gene therapy. Graeme: Gene therapy is, at this stage, experimental. COMM: She's undergone a course of chemotherapy and had her bone marrow harvested, and inserted back into her body. The treatment has been a roller-coaster of emotions for Nina and the family, but it's hoped that one day, she will live a normal life. Aga: After six months, there is a review, and this review has to say that her body is producing her own enzyme and she's just going to be a normal little girl who can go to school and play with other children.
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