1 March 2018 - Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments ...
19 February 2018 - Vertex has presented NHS England with an “innovative long-term framework proposal” that hopes to secure access ...
19 February 2018 - They have a deadly genetic disease their parents say is being halted by Brineura, but officials ...
12 February 2018 - NICE has published draft guidance that does not recommend the drug cerliponase alfa (Brineura; Biomarin) for children ...
11 January 2018 - A group of pharma companies and Duchenne UK are collaborating to improve the chances of new ...
20 December 2017 - The National Institute for Health and Care Excellence has rejected Clinuvel UK’s afamelanotide for treating erythropoietic ...
5 July 2017 - An agreement has been reached that means more people with a rare life threatening disease can be ...
29 June 2017 - Sanofi’s Cerdelga can now be funded on the NHS in England and Wales to treat type ...
14 June 2017 - Health technology assessment coverage recommendations differ across countries for the same drugs. Unlike previous studies, this study ...
12 June 2017 - The NICE it remaining steadfast in its position that Roche’s Esbriet should only be funded by ...
31 May 2017 - The NICE has issued a positive final appraisal determination recommending Cerdelga (eliglustat) as a first-line treatment ...
12 April 2017 - The UK government needs to adapt its drug and technology appraisal methods to prevent patients with rare ...
28 March 2017 - Nearly 200 rare disease patient groups have come together to call on NICE and NHS England ...
22 March 2017 - The UK’s public health watchdog is pressing ahead with plans to introduce a “dynamic upper limit” ...
14 March 2017 - French drug major Sanofi and its specialty care global business unit, Sanofi Genzyme, say they are ...