rare diseases

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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

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

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

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

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

Government lays out rare diseases plan

March 5, 2015
Sales and Marketing Department of Health, NHS, UK, government, howe, rare diseases

The UK government has set out new plans to improve the lives of patients with rare diseases by 2020. The …

News

Pfizer signs universities deal

May 12, 2014
Research and Development, Sales and Marketing AstraZeneca, Pfizer, academics, rare diseases

Pfizer has signed a five-year research deal with several top British universities to hunt for new treatments for rare disease. …

News

RPS: ‘Give pharma incentives’

May 8, 2014
Research and Development, Sales and Marketing NHS, RPS, rare diseases, royal pharmaceutical society

Incentives are urgently required to speed up the development of personalised medicines, according to a new report from the Royal …

News

EU regulator recommended 81 medicines in 2013

January 21, 2014
CHMP, EC, EU, FDA, US, rare diseases

Last year the Committee for Medicinal Products for Human Use (CHMP) recommended 81 medicines for marketing approval, compared with just …

News

Rare disease fund scrapped by NHS England

October 16, 2013
Research and Development, Sales and Marketing NHS England, fund, rare diseases

NHS England has pulled the plug on a £50 million fund to advance innovation in the investigation and treatment of …

News

Rare disease drugs ‘need separate budget’

September 3, 2013
Research and Development, Sales and Marketing NHS, NICE, rare diseases

A group of MPs in the UK are calling for a ring-fenced fund to pay for drugs for rare and …

News

Chiesi snaps up Zymenex

August 29, 2013
Sales and Marketing Chiesi, lamazym, rare diseases, zymenex

Chiesi Group is to acquire biopharma company Zymenex Holdings, allowing the Italian firm to enter the rare disease arena for …

News

Scotland fund for rare diseases

January 17, 2013
Sales and Marketing SMC, Scotland, rare diseases

The Scottish government is launching a £21 million fund in March to meet the costs of patients who require orphan …

News

Rare diseases – pharma’s new frontier

March 26, 2012
Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Big Pharma, CAPS, McConaghie, rare diseases

The last ten years has seen our understanding of rare diseases move ahead rapidly, often thanks to greater understanding of …

Feature