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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
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.
Latest news
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Senators attack Novartis over 'unconscionable' handling of Zolgensma data
At $2.1m, the rare disease gene therapy is the most expensive drug in American history. ... Earlier this year, Biohaven Pharmaceutical paid $105m for a rare paediatric priority review voucher.
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Sarepta investigating mistakenly submitted Duchenne therapy report
This study was a blinded, placebo-controlled trial investigating the use of a micro-dystrophin gene therapy candidate in people with DMD, a rare muscle-wasting disease that mostly affects boys ... Sarepta has been the lead in the DMD market, with some
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Who's calling the shots in European healthcare?
He also questions the ‘orphanisation’ of the industry’s pipeline, where US and EU initiatives have incentivised the sector toward developing more rare disease drugs, even though these might not be ... In the healthcare sector, innovation means
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Dupixent drives Sanofi again, but other new drugs are lagging
Genzyme posted a 22% increase in revenues to 2, 29bn, and while a lot of that was down to Dupixent there was also a 9% increase for its rare disease drugs.
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Otezla scores new indication as speculation over potential buyers grows
The drug is the first and only approved treatment option for patients with Behçet’s disease associated oral ulcers. ... The disease is a rare, chronic and multisystem inflammatory disease, with limited treatment options.
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Latest Intelligence
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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.
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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
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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:.
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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.
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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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Approximately 2 fully matching, plus 84 partially matching documents found.
Latest appointments
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Health comms agency emotive hires new cell and gene therapy leader
The agency says a large proportion of its work is now in the field of rare and ultra-rare disorders, where advanced therapies will play an important part in treatment protocols. ... product in a rare and previously untreatable eye disease.
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Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team
expertise as we shape our business for future growth with an unrelenting commitment to enhancing the lives of patients living with rare disease.
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BMS appoints new commercial leader, Davies joins Eisai and more
Sobi appoints head of technical operations. Rare disease specialist company Sobi has appointed Anne Marie de Jonge Schuermans to front its new technical operations organisation. ... Commenting on her appointment, Schuermans said: “Sobi has unique
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Abeona Therapeutics names CEO
Carsten Thiel joins the biopharma from Alexion. Rare genetic disease focused biopharma Abeona Therapeutics has named its chief executive officer in the form of Carsten Thiel. ... Steven Rouhandeh, executive chairman of Abeona, said: “As CEO, Carsten
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Therachon poaches Shire’s Aled Williams
Williams has more than 25 years’ experience in healthcare and pharmaceuticals and most recently he served as vice president and global strategy head for rare gastrointestinal diseases at Shire. ... Commenting on the recent appointment, Luca Santarelli,
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Approximately 0 fully matching, plus 31 partially matching documents found.
Latest from PMHub
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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.
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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
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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
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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. ... A Rare Disease UK report published in 2016 found that nearly half of the respondents who identified as undiagnosed had been waiting
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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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Approximately 9 fully matching, plus 53 partially matching documents found.
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- AZ, Merck’s Lynparza scores market-expanding clinical success
- Moving annual total (MAT)
- Roche undercuts price of Bayer cancer drug after winning FDA approval
- Leaked no-deal Brexit document forecasts widespread shortages
- FDA rejects Sarepta’s new Duchenne drug over safety fears
- Novo Nordisk shareholder revolt reaches Europe
- AstraZeneca buys FDA priority review voucher from Sobi
- Behaviour change – is there something missing?
- BeiGene gets speedy US review for lymphoma drug zanubrutinib
- NICE’s long-serving chief exec Sir Andrew Dillon to step down
- Pfizer goes big on gene therapy with $500m facility spend
- Empowered patients: shaking the foundations of healthcare
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We're passionate about helping our clients to make a real commercial impact. We achieve this through our unique approach to...
Latest intelligence
- Empowered patients: shaking the foundations of healthcare
- Precision medicine represents a new paradigm in healthcare.This new approach to treating and preventing disease views the patient holistically, analysing their genes, environment and lifestyle, and using this information to...
- A uniquely English genomic medicine service
- The UK National Health Service is developing one standardised approach to embedding precision medicine across the whole of England. Blue Latitude Health speaks to Dr Tom Fowler, Deputy Chief Scientist...
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- Data is the most valued commodity of the modern world. For P&P it's all about the application....
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