OGT 918 Study in Patients with Neuronopathic Gaucher Disease


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Following the OGT 918 trial for adult type 1 patients with Gauchers disease, a research study for patients with neuronopathic Gauchers disease will soon start at Great Ormond Street Hospital, London in collaboration with the National Institutes of Health in America, writes Tanya Collin-Histed


The study of OGT 918 in patients with neuronopathic Gauchers disease is an important step forward in the search for a treatment that may enter the brain (cross the blood brain barrier), and therefore may help the neurological symptoms of Gauchers disease.

In research studies, OGT 918 has been shown to inhibit the production of certain lipids (fatty material). It is hoped that by taking OGT 918, the storage of lipids will be reduced and the symptoms of neuronopathic Gauchers disease improved, in particular the neurological symptoms that other treatments are not able to treat effectively.

The current treatment in the management of Gaucher disease is enzyme replacement therapy but this therapy does not have a significant effect on the neurological symptoms of the disease.

The study, which will take place at Great Ormond Street Hospital in the UK and at the National Institutes of Health in the USA, is to evaluate the safety and effectiveness of OGT 918 together with enzyme replacement therapy compared with only enzyme therapy in the treatment of Type 3 Gauchers disease.

If you would like more information about the study taking place in either country, contact:

UK: Dr Ashok Vellodi, Great Ormond Street Hospital, London, telephone Elin Davies-Pope on: 0207 405 9200 bleep 803; (00 44 207 495 9200 from outside the UK).

USA: Dr Raphael Schiffman, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA, telephone: 1-800-411-1222. (001 800 411 1222 from outside the US)


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Source: Gauchers News April 2003.
© Copyright Gauchers Association 2003.