OGT 918, a substrate balance (deprivation) therapy, which is currently on trial for treatment of Gaucher's disease, has been re-named Vevesca. The drug was discussed at the European Working Group for Gaucher Disease meeting in Jerusalem on 6-9 September 2000 and at the National Gaucher Foundation Conference in Arlington, Virginia on 12-13 November 2000. OGT 918 was designated an Orphan Medicinal Product by the European Commission on 18 October 2000.
Susan Lewis reports on the progress so far:
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Prof Ari Zimran spoke about the research he has carried out into ozone therapy to aid pain control in people with Gaucher's disease, at the Israeli Gaucher's Association meeting held on 5 September 2000. This research was sponsored by the US National Gaucher Foundation and has been submitted for publication to the Journal of Pain and
Symptoms Management.
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About 180 individuals with Gaucher's disease and their families attended a two day Conference in Arlington, Virginia on 12-13 November 2000 to hear experts from the USA and Israel talk about the latest information on Gaucher's disease.
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Dr Paige Kaplan, who heads the Gaucher Center at the Children's Hospital of Philadelphia, spoke about the symptoms and problems of children with Gauchers Disease at the National Gaucher Foundation Conference on 12 November 2000.
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In July 2000 Healthcare at Home carried out a survey of patients with Gauchers disease who receive Cerezyme through their delivery and nursing service. Seventy eight responded stating where they were infused and whether they were satisfied with the delivery and, in some cases, the nursing service.
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Wojtek Oswiencinski has Gauchers disease, is 22 years old, a student and lives in Poland. His father, Pawel, is Chairman of the Polish Gauchers Association and Wojtek represents Poland at the European Gaucher Alliance. This is his personal story:
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Eleven countries were represented by their patient associations at the European Gaucher Alliance (EGA) meeting which took place in Jerusalem on 5-6 September 2000.
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The Israeli Gaucher Association Meeting was held on 5 September 2000 in Jerusalem to which delegates from the European Gaucher Alliance meeting were invited. Dr Gregory Grabowski spoke on 'Summarising 10 years of Enzyme Replacement Therapy' and Prof Ari Zimran on 'New Therapeutic Approaches to Gaucher Disease'.
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Patient associations for six different diseases were represented at two days of talks about the development of new enzyme replacement therapies and medical advances during a Congress for the Society for the Study of Inborn Errors of Metabolism held in Cambridge on 16-17 September. A special reception and dinner was hosted by Prof Timothy Cox and Mrs Joan Grantham on the Saturday evening.
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