Fabry Disease News and Research

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Fabry disease (also known as Fabry's disease, Anderson-Fabry disease, angiokeratoma corporis diffusum and alpha-galactosidase A deficiency) is a rare X-linked recessive (inherited) lysosomal storage disease, which can cause a wide range of systemic symptoms.

Further Reading

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Genzyme Corp. has announced the publication of a new study in The Proceedings of the National Academy of Sciences describing a novel preclinical gene therapy approach for treating Niemann-Pick disease.

24 May 2007

Stroke in young Fabry patients study

Stroke in young Fabry patients study

Stroke is the second leading cause of mortality worldwide and the most common cause of dementia in western countries.

15 Feb 2007

Drug shortfall as more than 5,000 conditions need medication

Drug shortfall as more than 5,000 conditions need medication

Only seven per cent of drug applications for treating people with rare diseases were approved in Europe between 2000 and 2004, despite the fact that there are currently more than 5,000 conditions needing medication.

9 Mar 2006

5,000 rare diseases need drugs, but Europe only approves a handful each year

5,000 rare diseases need drugs, but Europe only approves a handful each year

Only seven per cent of drug applications for treating people with rare diseases were approved in Europe between 2000 and 2004, despite the fact that there are currently more than 5,000 conditions needing medication.

6 Mar 2006

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