24 June 2022 - Adverum Biotechnologies today announced that the EMA has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment ...
24 June 2022 - EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Roctavian (valoctocogene roxaparvovec) ...
17 June 2022 - Givlaari (givosiran) is included in the high-cost protection with limited subsidy to reduce attacks in patients with ...
13 June 2022 - First and only FDA approved treatment demonstrating reversal in neuropathy impairment with subcutaneous administration once every three ...
2 June 2022 - Evidence judged to demonstrate that beti-cel provides net health benefit to patients with transfusion-dependent thalassaemia. ...
24 May 2022 - If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with haemophilia B. ...
20 May 2022 - CADTH does not accept new evidence demonstrating Spinraza's effectiveness and safety for treating spinal muscular atrophy – ...
20 May 2022 - First ever gene therapy directly administered into the brain. ...
17 May 2022 - The US FDA's top biologics regulator said the use of a “playbook” or platform approach ...
10 May 2022 - Amicus Therapeutics today announced that the U.S. FDA has extended the review period by 90 days ...
5 May 2022 - Report will be subject of CTAF meeting in November 2022; draft scoping document open to public ...
5 May 2022 - ICER evaluated the clinical and cost-effectiveness - as well as budgetary impact - of one of two ...
4 May 2022 - Zolgensma has the great honour of being the first medicine listed on the PBS with a ...
2 May 2022 - How should society handle new gene therapies for serious diseases, where the effect of the drugs is ...
13 April 2022 - Public comment period now open until 10 May 2022; requests to make oral comment during public ...