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rare disease

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

Pfizer goes big on gene therapy with $500m facility spend

Pfizer goes big on gene therapy with $500m facility spend

It also took a 15% stake in  Vivet Therapeutics and its lead programme for Wilson disease, a devastating rare, chronic and potentially life-threatening liver disorder.

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  • 25 Women Leaders in UK Healthcare 25 Women Leaders in UK Healthcare

    field. It has already produced some life-changing outcomes, giving one in four participants in the project with a rare disease a diagnosis for the first time, and providing potentially actionable ... 4. JAYNE SPINK . Rare disease patient champion.

  • 25 Women Leaders in UK Healthcare (part 2) 25 Women Leaders in UK Healthcare (part 2)

    23. DR LARISSA KERECUK. Rare disease leader. England’s groundbreaking 100, 000 Genomes Project hasn’t been a scientific endeavour achieved exclusively through lab technology – it has required thousands of rare ... A consultant paediatric

  • Rare disease trials Q&A Rare disease trials Q&A

    What are some solutions to these challenges that are most effective in rare disease studies? ... This approach must include the consideration of the following for all rare disease trials:.

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

    Rare disease drugs can also generate high revenues, said Garth Ringheim, Therapeutic Area Director, Clarivate Analytics. ... Patient-centricity is also core to Pfizer’s rare disease recruitment strategy, said Brownrigg.

  • Bluebird: on a mission to ‘recode’ the DNA of healthcare Bluebird: on a mission to ‘recode’ the DNA of healthcare

    Approved by the EMA in 2012, the rare disease treatment was priced at over 1m, and proved to be a commercial flop – partly because of its less than stellar efficacy, but ... to learn from its own mistakes with therapies for Huntington’s disease and

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  • A uniquely English genomic medicine service

    As Director of Public Health at Genomics England, he led the rare disease pilot phase of the 100, 000 Genomes Project. ... As new knowledge is generated, we can identify rare variants,” says Tom.

  • 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 ... In line with the Rare Disease Day theme, we took photos of our team

  • Porphyria Awareness Week – Round Up

    Porphyria Awareness Week has given the team at emotive an opportunity to show our support for those touched by this debilitating Rare Disease as well as our enthusiasm for working in ... Porphyria is a rare condition that is thought to affect around 1 in

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

    Children can be left facing an uncertain future and an unclear treatment pathway when they have a rare disorder. ... A   Rare Disease UK  report published in 2016 found that nearly half of the respondents who identified as undiagnosed had been waiting

  • After a busy first quarter, emotive welcomes five new team members

    With two exciting new EMEA and Global accounts added in Rare Disease and Advanced Therapies along with expanding existing client work, emotive is delighted to welcome five new recruits to the ... treatment of rare diseases.” said Dorothy at the end of

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