SMA Daily

Nusinersen Might Become the First Approved SMA Drug

AveXis Gains Orphan Drug Designation from the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy

Gene Therapy Stops or Stabilizes Spinal Muscular Atrophy in Animal Model

AveXis Doses First Patient in Gene Transfer Trial for Spinal Muscular Atrophy

AveXis Announces Opening of Enrollment for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

AveXis-BioLife Licenses Spinal Muscular Atrophy Gene Therapy Program