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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.

Orchard plans IPO as gene therapy pipeline advances

Orchard plans IPO as gene therapy pipeline advances

Orchard plans IPO as gene therapy pipeline advances. UK biotech seeks funds for expanded rare disease portfolio. ... It will also help bring forward its rapidly-expanding pipeline of rare disease candidates including ADA-SCID follow-up OTL-101, OTL-200

Latest news

  • Will Shire agree to extend Takeda takeover talks? Will Shire agree to extend Takeda takeover talks?

    Will Shire agree to extend Takeda takeover talks? UK rules mean Shire must agree to extend deadline. ... making a move for the Dublin-domiciled, UK-registered rare disease company.

  • GSK hands rare disease assets over to Orchard GSK hands rare disease assets over to Orchard

    GSK hands rare disease assets over to Orchard. The UK pharma group will take a 19.9% stake in its ex-rival. ... GlaxoSmithKline has decided the future of its rare disease business - and it lies outside the company.

  • emotive partners with Rare Disease UK for public awareness campaign emotive partners with Rare Disease UK for public awareness campaign

    emotive partners with Rare Disease UK for public awareness campaign. Will launch the campaign ahead of next month's Rare Disease Day. ... Partnering with emotive, Rare Disease UK hopes that the agency can bring creativity to their campaign.

  • Amicus gets NICE green light for Fabry drug Amicus gets NICE green light for Fabry drug

    The National Institute of Health and Care Excellence has given its blessing to Amicus' oral therapy for Fabry disease, a rare genetic disorder. ... The UK's cost-effectiveness regulator has published final guidance endorsing the use of Galafold

  • Pharma companies back Rare Disease Day Pharma companies back Rare Disease Day

    In the UK, Rare Disease Day 2014 is focusing on the implementation of the UK Strategy for Rare Diseases, launched last November. ... UK staff, in addition to taking part in the torch relay, will submit articles to the national Rare Disease Day supplement.

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Latest Intelligence

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in number and geographically spread,

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Accelerating diagnosis in rare disease. Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in

  • 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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