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Rare disease UK

This page shows the latest Rare disease UK news and features for those working in and with pharma, biotech and healthcare.

MHRA grants bluebird bio’s LentiGlobin gene therapy an ‘innovation passport’

MHRA grants bluebird bio’s LentiGlobin gene therapy an ‘innovation passport’

The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted bluebird bio’s potential sickle cell disease gene therapy a new ‘innovation passport’. ... In the UK, the rare genetic blood disorder affects approximately 15, 000 people.

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  • The trials and tribulations of rare opportunities The trials and tribulations of rare opportunities

    Jack Brownrigg, Medical Affairs, Rare Disease, Pfizer UK, told us clinical trials with fewer potential participants required more nuanced trial designs. ... Patient-centricity is also core to Pfizer’s rare disease recruitment strategy, said Brownrigg.

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Biopharmaceutical companies can drive effective partnerships and improve outcomes for all stakeholders and patients in the area of rare disease. ... Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare

  • A rare talent A rare talent

    As part of his efforts in this area Sebastian chairs the Rare Disease Strategy Group with UK industry body the ABPI. ... Making sense of change. From a newly enlarged company to new premises, to - perhaps - new HTA arrangements for rare disease

  • The distressing impact of rare diseases The distressing impact of rare diseases

    The company, which has a significant stake in the area, conducted online surveys in January among more than 1, 000 US/UK rare disease patients, carers, doctors, payers, government/institution representatives ... A UK patient with mixed connective tissue

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  • emotive supports Rare Disease Day 2019

    With a series of events and activities over the week leading up to Rare Disease Day on February 28th, we showed our support for stimulating greater awareness of Rare Diseases along ... To find out more about rare diseases, visit  Rare Disease UK. You

  • Learning about Rare Genetic Disorders on Undiagnosed Children’s Day

    26th April marks Undiagnosed Children’s Day; a day to celebrate and raise awareness for the 6, 000 children that are born annually in the UK with an unknown, rare genetic ... A   Rare Disease UK  report published in 2016 found that nearly half of the

  • Rare Disease Day 2019

    Today is #RareDiseaseDay and is an opportunity to highlight some truly unbelievable/frightening statistics around rare diseases. ... Did you know, for example, that over 3.5 million people in the UK suffer from a rare disease or that 95% of rare diseases

  • Rare Disease Day 2018: Five things you should know about rare diseases.

    Accessed February 2018. 7. Rare Disease UK. What is a rare disease? ... Accessed February 2018. What is a rare disease? Available at: .

  • emotive. ‘4817’ annual conference

    The first guest speaker of 4817 was Farhana Ali, from campaign group Genetic Alliance/Rare Disease UK. ... These were judged at the end of the second day, with the winning idea being worked up in preparation for a full presentation to the Rare Disease UK

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