AveXis

news

FDA approves first gene therapy for paediatric spinal muscular atrophy

29 May 2019 | By Victoria Rees (European Pharmaceutical Review)

The FDA has given approval to AveXis for the first treatment of paediatric spinal muscular atrophy (SMA).

news

AveXis gains EU Orphan Drug designation for gene therapy treatment in spinal muscular atrophy

24 June 2015 | By Victoria White

EMA grants Orphan Drug designation to AveXis' gene transfer therapeutic currently in clinical development for the treatment of spinal muscular atrophy...

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AveXis

FDA approves first gene therapy for paediatric spinal muscular atrophy

AveXis gains EU Orphan Drug designation for gene therapy treatment in spinal muscular atrophy