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VEXAS syndrome in men is more common than previously thought
A rare disease first identified in 2020 is much more common than first thought, say researchers at the University of Leeds investigating its origins.
VEXAS syndrome is a serious inflammatory condition which develops in men over 50, causing them to become very sick and fatigued, and can be fatal. It was originally thought to be rare, but a new study has identified genetic mutations which indicate that the disease is actually much more common.
The researchers developed a genetic test to identify patients who may have the disease, and now want to screen more people showing symptoms to understand exactly how common it is.
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A rare disease first identified in 2020 is much more common than first thought, say researchers at the University of Leeds investigating its origins.
VEXAS syndrome is a serious inflammatory condition which develops in men over 50, causing them to become very sick and fatigued, and can be fatal. It was originally thought to be rare, but a new study has identified genetic mutations which indicate that the disease is actually much more common.
The researchers developed a genetic test to identify patients who may have the disease, and now want to screen more people showing symptoms to understand exactly how common it is.
Advances in diagnosis and care have yielded significant improvements in childhood cancer survival rates in Europe, but the long-term side-effect burden in young people driven by the unlicensed use of adult cancer medicines often means the price of survival is high, scientists say.
Prescribing unlicensed drugs or the ‘off-label’ use of adult medicines for childhood cancer is largely the norm since the incidence of disease compared to adults is rare, which has disincentivised the biopharmaceutical industry from investing in paediatric research.
Somehow, it has been widely accepted that it is standard for children with cancer to be treated with off-label adult drugs, says Prof. Ruth Ladenstein, project coordinator of the European Reference Network on Paediatric Cancer and a member of the EU’s cancer mission board (see box below for more on the mission).
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No deal Brexit could have detrimental impact for four million people in UK living with a rare disease
Experts have warned that a ‘no deal’ Brexit will result in the exclusion of the UK from the 24 European Reference Networks (ERNs) that were established to improve the care of patients bearing the lifelong burden of a rare disease, which require highly specialised diagnosis and treatment.
Rare diseases are rare, and experts are rarer still. European Reference Networks were set up because no single country has the expertise or resources to cover all of the known rare diseases, which number in the thousands