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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

    To find out more about rare diseases, visit  Rare Disease UK. ... You can also find out about the great work of Medics 4 Rare Disease – including our partnership – on their  website and across social media.

  • 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

  • Improving healthcare for rare disease patients Improving healthcare for rare disease patients

    Accessed March 1, 2022. Rare Disease UK. Illuminating the Rare Reality; 2019. ... Accessed March 1, 2022. Rare Disease UK. Patient Empowerment.

  • 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.

    4. About 7, 000 types of rare disease are currently known to exist [10]. ... Available at: . Accessed February 2018. 6. Asbestos.com. Peritoneal mesothelioma. Available at: . Accessed February 2018. 7. Rare Disease UK. What is a rare disease?

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