Month: January 2019

January 9, 2019
Haemophilia gene therapy: we are not alone

The number of trials of cell and gene therapy carried out in the UK has increased by more than one-third in the past year, according to the Cell and Gene Therapy Catapult (, a London-based development agency that provides support for innovators in the field. The Catapult’s latest statistics show there are 85 clinical trials …

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Risk factors in children and young people with haemophilia

Concern about the possible risk of age-related disorders among people with haemophilia has been tempered by some evidence that many do not appear to be at greater risk than the population without bleeding disorders. Recent evidence from Turkey suggests some young people with haemophilia have cardiovascular risk factors – but no more than other people …

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Untreated bleeds are common

Patient diaries show that 40% of bleeds in people with haemophilia are not treated, according to a study presented at the 60th American Society of Hematology Annual Meeting in San Diego in December 2017. The study was part of the emicizumab development programme carried out by Genentech/Roche. Children and adults with haemophilia A used a …

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Acquired haemophilia A management remains a challenge

The best way to manage acquired haemophilia A in older people is unclear, according to a systematic review by specialists in France (Semin Hematol 2018;55:197-201). Their analysis of 80 publications describing 159 patients found a that a range of interventions was used and their effectiveness is difficult to assess. Mortality was high and depended on …

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