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rare diseases
This page shows the latest rare diseases news and features for those working in and with pharma, biotech and healthcare.
EFPIA-EURORDIS joint statement published on patient access to medicines for rare diseases
The statement contains a series of proposals to improve the lives of people living with rare diseases by addressing access barriers to transformative medicines. ... The EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases
Latest news
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UK government allocates up to £680m a year to two funds offering cutting-edge treatments
The IMF will provide potentially life-saving drugs for rare and genetic diseases, with the UK government pledging up to £680m per year to be divided up between the funds. ... For those living with rare conditions, approving treatments for rare disease
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New Pfizer initiative, ‘An Accord for a Healthier World’, launched to improve global health equity
This is how all global problems should be tackled.”. The company has also pledged to provide 23 medicines and vaccines to treat infectious diseases, certain cancers and rare and inflammatory diseases. ... These diseases result in almost one million
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SOLVE FSHD announces the appointment of Dr Eva Chin as its executive director
It was also shared that Chip Wilson, founder of SOLVE FSHD, has committed a personal pledge of $100m towards a cure for facioscapulohumeral muscular dystrophy (FSHD), a rare disease he was ... quality of life for those affected by rare neuromuscular
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Novo Nordisk and Flagship Pioneering to collaborate on new research programmes
Using Flagship’s innovative bioplatforms from a portfolio of 41 companies, the partnership will address scientific challenges within rare and cardio-metabolic diseases by creating research programmes to specifically target these ... This is a new and
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AstraZeneca’s Alexion agrees on $775m settlement with Chugai for Ultomiris
AstraZeneca’s (AZ) rare disease group subsiduary Alexion has reached a settlement agreement with Chugai Pharmaceutical Co (Chugai). ... of people affected by rare diseases.”.
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Approximately 131 fully matching, plus 300 partially matching documents found.
Latest Intelligence
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How understanding the rare disease patient’s lived experience creates better health communications
Understanding the lived experience – a real life example. Successful healthcare communication within rare diseases depends on forming a deep understanding of the interwoven HCP and patient pathways. ... The film highlights the fact that one in 20
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A creative awakening – how pharma is bringing innovation to the patient journey
Patient populations, particularly those with rare diseases, network with each other via the internet, and HCPs connect with each other and their patients digitally, but pharma has not really caught up ... Hall added: “Digital and technology advances
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Listen, learn and lift off
He added: “Pharma has been getting better at this and it will become even more important as industry focuses more on rare diseases. ... That has completely changed and we’re now launching regularly thanks to huge advancements in rare diseases, which
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Understanding the ‘real world’ starts and ends with the patient
Rare disease R&D hinges on the ‘real world’. Patient engagement in rare diseases is imperative. ... However, providing data in rare diseases is rarely a ‘slam dunk’, as the generation of RWE comes from multiple sources.
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Pre-launch planning and DSLs – the new ways to create launch excellence in pharma
One trend that we’re witnessing in industry data, and from anecdotal evidence from our clients, is that more specialised medicines are being developed and there is more investment into rare ... diseases and niche indications, and increasingly segmented
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Approximately 17 fully matching, plus 116 partially matching documents found.
Latest appointments
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Mark Caulfield departs from Genomics England to take on CEO role at Barts Life Sciences
First launched in 2012, the 100, 000 Genome Project had ambitious plans to map the genomes of NHS patients to deliver treatments for those with rare inherited diseases and cancer.
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Azafaros expands executive team with three senior hires
While at Sanofi, she served as head of rare diseases, Central and Eastern Europe – in this role, she led all commercial activities for rare disease-related programmes in those regions.
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Porter Novelli adds Amber Tovey to London health team
In her new role at Porter Novelli, Tovey will work across a global pharmaceutical client portfolio that includes neurology, rare diseases and HIV.
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New hires at Crescendo, Healx and Galecto
Hall (pictured left) has over 25 years of experience in drug development, with the last decade spent focused on rare diseases. ... He co-founded the rare disease patient group support charity Findacure with Nick Sireau, the advisor to and the inspiration
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New hires at bluebird bio, BioNTech and Provention Bio
Jason's extensive commercial experience, including the planning, preparation and execution of product launches in rare diseases and nascent markets, will be critical as we work to bring teplizumab to type
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Approximately 1 fully matching, plus 60 partially matching documents found.
Latest from PMHub
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emotive supports Rare Disease Day 2019
On 20th February, we attended ‘The Unusual Suspects: Rare Diseases in Everyday Medical Care” at the Royal Society of Medicine, hosted by Medics 4 Rare Diseases (M4RD). ... emotive is proud to be working in rare diseases for a number of clients.
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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. ... 3) The child might have an array of symptoms that are in fact an unusual presentation of a known rare condition.
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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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“My first six months in medical communications” – views from an emotive graduate recruit, Claire
It will help raise awareness of rare diseases amongst medical students and doctors so it will have a really positive impact.”. ... What surprised me was the fast pace and the vast range of diseases (especially rare diseases) that we work on – a lot
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Three amazing women at emotive talk about what they love about medical communications
Everyone is passionate about their work. Working in rare diseases, where there are so many exciting new advances, I feel good about what we do. ... emotive is an award-winning healthcare communications agency working with leading global life sciences
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Approximately 30 fully matching, plus 115 partially matching documents found.
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