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rare diseases

Showing 15 posts of 24 posts found.

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Saniona appoints Rami Levin as President and Chief Executive Officer

January 8, 2020
Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing appointments, rare diseases

Saniona has announced that Rami Levin will take over as the Chief Executive Officer of the company as it seeks …

Appointment
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Roche scores two EU approvals in haemophilia and rare autoimmune disease

March 15, 2019
Medical Communications EC, EU, Europe, Roche, haemophilia, rare diseases

The European Commission (EC) has approved Roche’s haemophilia treatment Hemlibra (emicizumab). The approval comes as the EC approved Roche’s MabThera …

News
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China to establish national rare disease network

February 19, 2019
Manufacturing and Production China, NHC, healthcare, rare diseases

China is set to establish a national collaborative network of hospitals for rare disease diagnosis and treatment to promote early …

News
pharmafile_autumn_2018

The Autumn 2018 edition of Pharmafile is available to read online now!

December 18, 2018
Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing immunology, infectious diseases, pharma, pharmafile, rare diseases

The Autumn 2018 edition of Pharmafile, our long-running bi-annual guide to the latest trends, projections and thought leadership positions in …

News
astrazeneca_plaque

FDA approves AstraZeneca’s Lumoxiti for hairy cell leukaemia (HCL)

September 17, 2018
Sales and Marketing AstraZeneca, HCL, Lumoxiti, leukaemia, rare diseases

AstraZeneca’s Lumoxiti has been approved for use for the treatment of relapsed or refractory hairy cell leukemia (HCL)in adults who …

News
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Global orphan and rare dermatology market set to exceed $6 billion by 2024

August 31, 2018
Medical Communications Orphan Drugs, analysis, dermatology, market reports, rare diseases

The global orphan and rare dermatology premium products market is expected to grow rapidly in the next half a decade …

News
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FDA approves RNA therapy for rare genetic disease

August 13, 2018
Sales and Marketing FDA, Onpattro, Orphan Drugs, RNA, rare diseases

The US Food and Drug Administration has approved the RNA-based therapy Onpattro, for the treatment of the rare genetic disease …

News
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The need for flexible appraisal of rare disease medicines

March 15, 2018
Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Amryt Pharma, feature, pharma, rare disease, rare diseases

Recent data has shown that England is, on average, slower than Germany, France, Spain and Italy in making orphan medicinal …

Feature

Taking science and making people feel connected to it

August 21, 2017
Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Pfizer, biotech, drugs, pharma, pharmaceutical, rare diseases

Owen Marks, Country Head for the Rare Disease Unit at Pfizer UK, discusses his career so far and what drives …

Feature
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Shire’s drug picked up from Baxalta given fast track designation

March 23, 2017
Research and Development, Sales and Marketing Shire, rare diseases

Shire has announced that its drug, SHP655, has received a fast track designation based on pre-clinical trials and positive results …

News
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Horizon and Fulcrum Therapeutics form CRISPR collaboration

September 6, 2016
Manufacturing and Production, Research and Development Horizon Discovery, crispr, fulcrum, fulcrum therapeutics, gene editing, horizon, rare diseases

Horizon Discovery has announced that it has entered into a partnership with Fulcrum Therapeutics to utilise Horizon’s CRISPR-based screening platform …

News
findacure

Drug repurposing can help tackle Rare Diseases, says charity

February 29, 2016
Medical Communications, Research and Development, Sales and Marketing rare diseases

On Rare Disease Day 2016, UK charity Findacure says existing drugs may hold the key to new treatments and repurposing …

News

More evidence required for Alexion’s Kanuma, says NICE

February 18, 2016
Medical Communications, Research and Development Alexion, Kanuma, LAL-D, rare diseases

NICE says it needs additional trial data before it can recommend Alexion’s enzyme replacement therapy Kanuma (sebelipase alfa) for the …

News
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Novartis forms Mereo as UK biotech spin-off

July 29, 2015
Research and Development Mereo, Novartis, biotech, rare diseases

Novartis has formed a new UK biotech company by selling three investigational drugs in exchange for shares in the new …

News
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Overcoming barriers in rare diseases

June 22, 2015
Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing BIA, NHS, Shire, bates, government, nps, rare diseases

In recent months the focus on the need to develop new drugs and fund new treatments for rare diseases has …

Feature

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