1 March 2021 - The treatment delays permanent ventilation of the children and extends life. In addition, the children develop more ...
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...
16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has ...
Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy Zolgensma, which is used to treat toddlers with ...
26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...
19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...
15 January 2021 - After correcting an input to the cost-effectiveness model, ICER’s health-benchmark price benchmark range for nadofaragene firadenovec has ...
21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...
19 December 2020 - Reign Johnston is a happy baby who enjoys painting as much as playing with toys. His green ...
18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...
18 December 2020 - The recommendations from the November 2020 PBAC meeting are now available. ...
16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective ...
11 December 2020 - The promise of gene therapy is to cure diseases associated with faulty or missing genes. There’s enormous ...
3 December 2020 - enGene announced today that the U.S. FDA has granted fast track designation to enGene for EG-70, the ...
19 November 2020 - Oxlumo is the first therapeutic approved for the treatment of primary hyperoxaluria type 1, and the ...