Two-year-old boy ‘cured’ of rare disease with pioneering stem cell treatment after being given weeks to liveBy Gemma Mullin, Digital Health ReporterA TWO-year-old boy who was given just weeks to live has been "cured" of a rare disease after medics used a pioneering technique to treat him.
He was being treated with an experimental drug at Great Ormond Street Hospital but his parents Paolo and Cristina were told it may become ineffective after five weeks. Alex will still need to have regular check-ups, but Dr Locatelli said the youngster was "ready to resume the life that all children of his age should have."Desperate appeal
His parents explained that the condition affects "only 0.002 per cent of children" and that "50 per cent of children affected by this disease die within the first month".Haemophagocytic lymphohistiocytosis is a rare immune disorder where the body reacts inappropriately to a ‘trigger’, usually an infection.
They include a skin rash, raised temperature and swollen liver, spleen and lymph glands. There may be anaemia, infection or bruising and bleeding. If the brain is affected, a child may show symptoms such as seizures, ataxia or drowsiness.
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