AveXis – pharmaceutical daily

October 7, 2019 Off

AveXis has early encouraging results for spinal muscular atrophy patients treatment

Novartis’ company Avexis said that it’s Phase 1/2 Srong study for intrathecal (IT) administration of AVXS-101, showed that patients aged 2 to 5, with spinal muscular atrophy (SMA) Type 2 achieved a mean increase of 5.9 points from baseline in HFMSE scores, nearly double the clinically meaningful threshold, at a mean duration of follow-up time of 9.3 months.

December 3, 2018 Off

FDA accepts Novartis’s Biologics License Application for Zolgesma

By Dino Mustafić

Novartis’s Biologics License Application (BLA) for Zolgensma, designed to tackle the genetic root cause of spinal muscular atrophy (SMA) Type 1, a deadly neuromuscular disease, was accepted by the U.S. Food and Drug Administration (FDA).

April 9, 2018 Off

Swiss drugmaker Novartis buys US-based gene therapy company for $8.7 billion

By Dino Mustafić

Novartis’s acquisition of the US-based Nasdaq-listed clinical stage gene therapy company AveXis for $218 per share, for total of 8.7…