Type 1 spinal muscular atrophy (SMA) babies treated with AveXis’ gene therapy candidate AVXS-101 continue to achieve unprecedented motor milestones and do not require breathing or nutritional support, according ... Read more
AveXis’ expects to start a Phase 3 clinical trial of its spinal muscular atrophy (SMA) gene therapy AVXS-101 in Europe in May. The STR1VE-EU trial (NCT03461289) is expected to include up to ... Read more
A broad range of clinical studies into AveXis’s gene therapy AVXS-101 is likely to decide whether it is indeed a “transformative” treatment for babies and children with spinal muscular atrophy (SMA) types ... Read more
The gene therapy developer AveXis will start a pivotal clinical trial of AVXS-101 for people with SMA type 1.
U.S. Food and Drug Administration officials agreed to the trial after AveXis submitted information the ... Read more
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