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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.

Celgene says no to Mereo’s cancer drug etigilimab

Celgene says no to Mereo’s cancer drug etigilimab

UK biotech will focus on rare disease drugs. Mereo BioPharma has lost Celgene as a partner for anti-TIGIT drug etigilimab, one of two drugs acquired when Mereo merged with OncoMed ... MPH-966) for alpha-1 antitrypsin deficiency, while looking for

Latest news

  • How to fix a problem like Orkambi? Solutions suggested ahead of showdown How to fix a problem like Orkambi? Solutions suggested ahead of showdown

    The Commons’ committee has published written submissions from a number of stakeholders, ranging from the UK biotech association the BIA, rare disease campaign group Genetic Alliance, David Turner, a barrister, CF ... They say other countries have

  • UK launches National Genomic Healthcare strategy UK launches National Genomic Healthcare strategy

    New initiative unveiled to coincide with Rare Disease Day. The UK government has announced plans to further its goal of becoming a world-leader in bringing genomics into everyday healthcare. ... For patient groups like Rare Disease UK and pharma companies

  • ‘Inflexible’ NICE blocking access to rare disease drugs ‘Inflexible’ NICE blocking access to rare disease drugs

    The report has won the endorsement of Genetic Alliance UK, an umbrella organisation which represents many of the UK’s rare disease charities. ... UK. “This system will continue to fail people living with rare diseases until the fundamental challenges

  • 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. UK gene therapy specialist Orchard Therapeutics has filed registration documents for an initial public offering, just weeks after its latest $150m private ... It will also help bring forward its

  • 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. Shire is mulling over Takeda’s improved £42.4bn ($60 bn) - and must decide by 5pm UK time tomorrow whether or not ... making a move for the Dublin-domiciled, UK-registered rare disease company.

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Latest Intelligence

  • 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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Latest from PMHub

  • 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. 8.RareDiseaseDay.org. 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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