22 June 2021 - Following the FDA’s recent decision to give the green light to aducanumab, the first treatment approved ...
23 June 2021 - Beth Vanstone, mother of local Cystic Fibrosis Warrior Madi Vanstone and director of the CF Get ...
18 June 2021 - Orphazyme today announced it has received a complete response letter from the U.S. FDA following its ...
2 June 2021 - NICE has today published draft guidance for public consultation which does not recommend risdiplam (Evrysdi, Roche) for ...
31 May 2021 - Conventional appraisal and reimbursement processes are being challenged by the increasing number of rare disease treatments with ...
28 May 2021 - New drug application granted priority review status, with a decision anticipated on 30 November 2021. ...
24 May 2021 - Builds on Priority Medicines (PRIME) designation recently granted by the EMA recognising the unmet medical needs of ...
14 May 2021 - Empaveli was superior to Soliris for the change from baseline in haemoglobin level at Week 16 in ...
12 May 2021 - Fulcrum Therapeutics today announced that the U.S. FDA has granted fast track designation to losmapimod for ...
4 May 2021 - Gilead’s antibody-drug conjugate Trodelvy (sacituzumab govitecan) has won the Ministry of Food and Drug Safety’s designation ...
28 April 2021 - New indication includes people ages 12 years and older who have one copy of the F508del mutation ...
27 April 2021 - After weeks of controversy, Biogen has agreed to provide an experimental drug for combating ALS to ...
19 April 2021 - Alnylam has submitted a new drug application to the U.S. FDA for the approval of vutrisiran for ...
19 April 2021 - Citrine Medicine and Sinopharm Group today announced a strategic partnership to accelerate the commercialisation of rare ...
9 April 2021 - Masterton's Allyson Lock feels she is not part of the "team of five million" after her ...