16 February 2022 - Initiation of Phase 1/2 clinical trial expected in the second half of 2022. ...
4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment ...
4 February 2022 - The NHS has struck a confidential deal for what's thought to be the most expensive drug ...
1 February 2022 - Experience from clinical practice with Zolgensma, a gene therapy against certain forms of spinal muscular atrophy in ...
24 January 2022 - Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least ...
18 January 2022 - FDA PDUFA goal dates for both therapies extended by three months. ...
10 January 2022 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation for 4D-125 for treatment ...
24 December 2021 - The Public Summary Documents (positive recommendations and subsequent decisions not to recommend) from the September 2021 PBAC ...
17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, ...
15 December 2021 - CSL Behring today announced that the CHMP, the chief scientific body of the EMA accepted its ...
14 December 2021 - Today the EU Parliament passed the EU-HTA regulation for a joint health technology assessment, which includes, ...
24 November 2021 - Report will be subject of New England CEPAC meeting in June 2022; draft scoping document open ...
22 November 2021 - FDA set PDUFA date of 20 May 2022. ...
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...
3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its ...