AVXS-101 May Be ‘Transformative’ Breakthrough for SMA Type 1, Cure SMA President Says
AveXis‘ gene therapy AVXS-101, now under review for approval to treat infants with spinal muscular atrophy (SMA) type 1 in the U.S. and Europe, may be truly “transformative,” said Kenneth Hobby, president of the patient advocacy group Cure SMA. Trial data in this patient group “shows that a one-time dose of…