Neurological Symptoms and Therapy

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Dr Bruno Bembi looks after 45 patients with Type 1 Gauchers disease at his hospital, Istituto per l'Infanzia Burlo Garofolo, in Trieste. He also has five Type 3 patients and five Type 2.

Type 2

'Two years ago, a Type 2 baby was treated with Ceredase infused directly into the brain via the cerebral ventriculum for 10 months,' reported Dr Bembi. Therapy was then stopped. The child's condition has remained stable, responding to its parents although seriously retarded, nearly two years later. Dr Bembi now proposes to treat another Type 2 baby age 5 months. Update: Dr Bembi treated the child for 3 months and then stopped. 'Progress was noted but not enough for normal life,' he said.

Type 3

Of the five Type 3 patients on enzyme replacement therapy whom Dr Bembi is treating in Trieste, three are progressing well with no further neurological deterioration.

The other two suffer from more severe neurological problems. However one of them aged 20 years has been on therapy for three years, feels good and is able to hold down a job. The other suffers from epilepsy but also works and is in a good mental condition.

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Source: Gauchers News September 1996

© Copyright Gauchers Association 1996