14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and ...
23 August 2021 - The FDA and the drug industry have jointly agreed to create several new pilot programs aimed ...
30 June 2021 - Phase I/II clinical trial in patients with cardiomyopathy associated with Friedreich’s ataxia expected to initiate in 2021. ...
28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...
22 June 2021 - Following the FDA’s recent decision to give the green light to aducanumab, the first treatment approved ...
18 June 2021 - Orphazyme today announced it has received a complete response letter from the U.S. FDA following its ...
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 ...
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 ...
11 March 2021 - Denali Therapeutics today announced that the U.S. FDA has granted fast track designation to ETV:IDS (DNL310) for ...
1 March 2021 - Rare Disease Day is a time to reflect on both the progress that has been made, and ...
26 January 2021 - Carbaglu is first and only FDA approved medication for hyperammonaemia associated with these rare conditions. ...