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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.
Landscape review reveals £1.1bn of UK funding for rare diseases
Since 2021, the NIHR and MRC have invested £14m in the UK Rare Disease Research Platform and LifeArc has invested £40m in a rare disease translational challenge. ... Professor Lucy Chappell, chief executive of NIHR, said: “This report is the first
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LifeArc launches rare disease programme with £100m investment
One in 17 people will be affected by a rare disease at some point in their lives, amounting to around 3.5 million people in the UK. ... The new UK Rare Disease Research Platform is set to bring together expertise from across the UK rare disease research
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NIHR and MRC establish UK Rare Disease Research Platform with £14m investment
The platform brings together expertise from across the UK rare disease research system. ... The new UK Rare Disease Research Platform is set to bring together expertise from across the UK rare disease research system to foster new and innovative
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Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients
The rare genetic disorder affects around one in every 40, 000 people in the UK. ... Affecting approximately one in every 40, 000 people in the UK, Pompe disease is a rare genetic disorder caused by low levels of the enzyme acid alpha-glucosidase,
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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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UK introduces Rare Diseases Framework to improve the lives of people with rare diseases
According to Rare Disease UK, one in 17 people will be affected by a rare disease at some point during their lifetime, with 3.5 million people in the UK and ... Helping patients across the UK to get a final diagnosis for a rare disease faster.
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Approximately 2 fully matching, plus 21 partially matching documents found.
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LifeArc to launch rare disease centres across the UK
One in 17 people will be affected by a rare disease at some point in their lives, amounting to around 3.5 million people in the UK. ... One in 17 people will be affected by a rare disease at some point in their lives, amounting to around 3.5 million
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How AI is finally helping rare diseases gain more than just attention
Social Care will implement the UK Rare Diseases Framework, which has been designed to improve the lives of those living with rare diseases. ... In fact, there are over 3.5 million people in the UK alone living with a rare disease, and as many as one in
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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.
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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
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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
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Approximately 0 fully matching, plus 6 partially matching documents found.
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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.
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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
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Medscape Education Global
Medscape are powering education and disease state awareness in infectious diseases at #IDWeek 2023. ... Medscape Education team up with The Guardian to launch a Rare Disease campaign in the UK.
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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.
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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
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- Pfizer’s RSV vaccine granted MHRA approval to protect infants and older adults
- Novavax’s updated COVID-19 vaccine granted Emergency Use Listing by WHO
- FDA to investigate risk of T-cell malignancy from CAR-T cell immunotherapies
- Boehringer Ingelheim and IBM announce AI antibody drug discovery partnership
- New blood test to reduce heart attacks and death rates in A&E heart patients
- Clairvoyant commercialisation
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